期刊名称：HUMAN GENE THERAPY CLINICAL DEVELOPMENT
 期刊简介(About the journal)
投稿须知(Instructions to Authors)
编辑部信息(Editorial Board)
About the journal
Human Gene Therapy
Editor-in-Chief: James M. Wilson, MD, PhD
Deputy Editor: Thierry VandenDriessche, PhD
Human Gene Therapy Editor: Mark A. Kay, MD, PhD
Methods Editor: Hildegard Büning, PhD
Clinical Development Editor: Barry J. Byrne, MD, PhD
Human Gene Therapy Clinical Development
The leading journal for publishing data relevant to the regulatory review and commercial development of cell and gene therapy products.
Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.
The Journal is divided into three parts. Human Gene Therapy, the flagship, is published 12 times per year. HGT Methods, a bimonthly journal, focuses on the applications of gene therapy to product testing and development. New in 2013, HGT Clinical Development, a quarterly journal, serves as a venue for publishing data relevant to the regulatory review and commercial development of cell and gene therapy products.
Complete HGT Program coverage includes:
- Improvements in vector developments
- Delivery systems
- Cell therapy
- Small nucleic acid therapeutics, including RNAi
- Clinical trials and animal models
- Clinical protocols
- Pre-clinical animal and in vitro studies to assess the safety of gene and cell therapy products used to support clinical applications
- Clinical trials including those in which the results are confirmatory or negative
- Topical issues related to the commercial development of gene and cell therapy products
Human Gene Therapy was voted one of the most influential journals in Biology and Medicine over the last 100 years by the Biomedical & Life Sciences Division of the Special Libraries Association.
Human Gene Therapy, HGT Methods, and HGT Clinical Development are under the editorial leadership of Editor-in-Chief James M. Wilson, MD, PhD, University of Pennsylvania School of Medicine, Gene Therapy Program, Department of Pathology and Laboratory Medicine; Deputy Editor Thierry VandenDriessche, PhD, Free University of Brussels (VUB); Human Gene Therapy Editor Mark A. Kay, MD, PhD, Division of Human Gene Therapy, Departments of Pediatrics and Genetics, Stanford University School of Medicine; Methods Editor Hildegard Büning, PhD, University of Cologne; Clinical Development Editor Barry J. Byrne, MD, PhD, Powell Gene Therapy Center, University of Florida, College of Medicine, and other leading investigators. View the entire editorial board.
Audience: Geneticists, medical geneticists, biological scientists, microbiologists, molecular biologists, virologists, experimental researchers, and experimental medicine specialists, among others.
Human Gene Therapy and HGT Methods provide “Instant Online” publication 72 hours after acceptance
This subscription includes:
Read Online |
Editor-in-Chief: James M. Wilson, MD, PhD
ISSN: 1043-0342 • Monthly • Online ISSN: 1557-7422
Tables of Content • Read a Free Issue
Editorial Board • Author Information • Subscribe
Human Gene Therapy is the definitive peer-reviewed rapid-publication journal covering all aspects of human gene therapy. The Journal publishes scientific papers on original investigations into the transfer and expression of genes in mammals, including humans with in-depth coverage of DNA, RNA, and cell therapies. Improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, are covered. |
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Read Online |
Editor-in-Chief: James M. Wilson, MD, PhD
ISSN: 1946-6536 • Bimonthly • Online ISSN: 1946-6544
Editorial Board • Author Information • Subscribe
Human Gene Therapy Methods (HGT Methods) is an expansion of the flagship Human Gene Therapy, publishing technological advances in cell and gene therapy that promote the development of gene therapy products into successful therapeutics. The Journal breaks new ground as the first to exclusively focus on the applications of gene therapy to product testing and development. |
|
Read Online |
Editor-in-Chief: James M. Wilson, MD, PhD
ISSN: 1043-0342 • Quarterly • Online ISSN: 1557-7422
Editorial Board • Author Information • Subscribe
Human Gene Therapy Clinical Development publishes data relevant to the regulatory review and commercial development of cell and gene therapy products. Pre-clinical papers focus on pharmacology/toxicity/bio-distribution experiments used in the review of proposed clinical trials and will contain sufficient information to allow referencing between products within the same technology platform when appropriate. |
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About This Publication:
Human Gene Therapy
Editor-in-Chief: James M. Wilson, MD, PhD
Deputy Editor: Thierry VandenDriessche, PhD
Human Gene Therapy Editor: Mark A. Kay, MD, PhD
Methods Editor: Hildegard Büning, PhD
Clinical Development Editor: Barry J. Byrne, MD, PhD
ISSN: 1043-0342 • Published 22 issues annually • Online ISSN: 1557-7422 Current Volume: 25
Latest Impact Factor* is 3.623
*2013 Journal Citation Reports® published by Thomson Reuters, 2014
Featured Press Releases and Editorial Content
- Pioneer Award Winners Katherine High, Amit Nathwani, Arthur Nienhuis, and Andrew Davidoff Honored for Advances in Hemophilia Gene Therapy
- Read the Perspective by Dr. High
- Read the Perspective by Drs. Nathwani, Nienhuis, and Davidoff
- Pioneer Award Recipients Zelig Eshhar, PhD and Carl H. June, MD, PhD Honored for Their Research on T-Cell Engineering for Cancer Immunotherapy
- Read the Perspective by Dr. Eshhar
- Read the Perspective by Dr. June
- Long-term Treatment Success Using Gene Therapy to Correct a Lethal Metabolic Disorder ...Read More
- Pioneer Award Recipients Robin Ali, PhD, Jean Bennett, MD, PhD, and William Hauswirth, PhD, Honored for Their Research on Gene Therapy in Eye Disorders
- Read the Perspective by Dr. Bennett
- Read the Perspective by Dr. Hauswirth
- Malcolm K. Brenner, MD, PhD Receives Pioneer Award for Advances in Gene-Modified T Cells Targeting Cancer...Read More
- Splice-Switching Oligonucleotide Therapeutics Is Promising New Method for Editing Gene Transcripts...Read More
- Christof von Kalle, MD, PhD Receives Pioneer Award for Contributions to Efficient Vector Integration in Human Gene Therapy...Read More
- Frederic Bushman, PhD Receives Pioneer Award for Advancing Therapeutic Gene Delivery Methods...Read More
- Gene Therapy Extends Survival in an Animal Model of Spinal Muscular Atrophy...Read More
- Novel RNAi Therapy Silences Mutated Huntington's Disease Gene and Reduces Symptoms...Read More
- James Wilson, MD, PhD Receives Pioneer Award for Research on Gene Therapy and AAV Gene Delivery Systems...Read Now
- Pioneer Award Recipients Marina Cavazzana and Adrian Thrasher Recognized for Advancing Gene Therapy to the Clinic for Immunodeficiency Disorders...Read Now
- Gene Therapy for Lysosomal Storage Disease Shown to Be Safe and Well Tolerated, with Promising Results...Read Now
- Exclusive David Gancberg Article in Human Gene Therapy on European Commission Funding 2014–2015...Read Now
- Joseph Glorioso, PhD, Receives Pioneer Award for Engineering Herpes Simplex Virus Gene Delivery Systems...Read Now
- Ronald Crystal, MD, Receives Pioneer Award for Developing First In Vivo Adenoviral Gene Delivery Vector...Read Now
- Top 12 Pioneer Awards for Seminal Work in Gene and Cell Therapy Selected by Blue Ribbon Panel; Essays to be Published in Human Gene Therapy...Read Now
- Special Tribute Issue for Sonia Skarlatos, PhD
Overview
Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.
The Journal is divided into three parts. Human Gene Therapy, the flagship, is published 12 times per year. HGT Methods, a bimonthly journal, focuses on the applications of gene therapy to product testing and development. New in 2013, HGT Clinical Development, a quarterly journal, serves as a venue for publishing data relevant to the regulatory review and commercial development of cell and gene therapy products.
Complete HGT Program coverage includes:
- Improvements in vector developments
- Delivery systems
- Cell therapy
- Small nucleic acid therapeutics, including RNAi
- Clinical trials and animal models
- Clinical protocols
- Pre-clinical animal and in vitro studies to assess the safety of gene and cell therapy products used to support clinical applications
- Clinical trials including those in which the results are confirmatory or negative
- Topical issues related to the commercial development of gene and cell therapy products
Human Gene Therapy was voted one of the most influential journals in Biology and Medicine over the last 100 years by the Biomedical & Life Sciences Division of the Special Libraries Association.
Human Gene Therapy, HGT Methods, and HGT Clinical Development are under the editorial leadership of Editor-in-Chief James M. Wilson, MD, PhD, University of Pennsylvania School of Medicine, Gene Therapy Program, Department of Pathology and Laboratory Medicine; Deputy Editor Thierry VandenDriessche, PhD, Free University of Brussels (VUB); Human Gene Therapy Editor Mark A. Kay, MD, PhD, Division of Human Gene Therapy, Departments of Pediatrics and Genetics, Stanford University School of Medicine; Methods Editor Hildegard Büning, PhD, University of Cologne; Clinical Development Editor Barry J. Byrne, MD, PhD, Powell Gene Therapy Center, University of Florida, College of Medicine, and other leading investigators. View the entire editorial board.
Audience: Geneticists, medical geneticists, biological scientists, microbiologists, molecular biologists, virologists, experimental researchers, and experimental medicine specialists, among others.
Human Gene Therapy and HGT Methods provide “Instant Online” publication 72 hours after acceptance
This subscription includes:
Read Online |
Editor-in-Chief: James M. Wilson, MD, PhD
ISSN: 1043-0342 • Monthly • Online ISSN: 1557-7422
Tables of Content • Read a Free Issue
Editorial Board • Author Information • Subscribe
Human Gene Therapy is the definitive peer-reviewed rapid-publication journal covering all aspects of human gene therapy. The Journal publishes scientific papers on original investigations into the transfer and expression of genes in mammals, including humans with in-depth coverage of DNA, RNA, and cell therapies. Improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, are covered. |
|
Read Online |
Editor-in-Chief: James M. Wilson, MD, PhD
ISSN: 1946-6536 • Bimonthly • Online ISSN: 1946-6544
Editorial Board • Author Information • Subscribe
Human Gene Therapy Methods (HGT Methods) is an expansion of the flagship Human Gene Therapy, publishing technological advances in cell and gene therapy that promote the development of gene therapy products into successful therapeutics. The Journal breaks new ground as the first to exclusively focus on the applications of gene therapy to product testing and development. |
|
Read Online |
Editor-in-Chief: James M. Wilson, MD, PhD
ISSN: 1043-0342 • Quarterly • Online ISSN: 1557-7422
Editorial Board • Author Information • Subscribe
Human Gene Therapy Clinical Development publishes data relevant to the regulatory review and commercial development of cell and gene therapy products. Pre-clinical papers focus on pharmacology/toxicity/bio-distribution experiments used in the review of proposed clinical trials and will contain sufficient information to allow referencing between products within the same technology platform when appropriate. |
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The Official Journal of nine international societies:
Indexed/Abstracted in:MEDLINE; PubMed; PubMed Central; Current Contents®/Life Sciences; Science Citation Index Expanded; Science Citation Index®; Biotechnology Citation Index®; Biological Abstracts; BIOSIS Previews; Journal Citation Reports/Science Edition; Derwent Drug File; EMBASE/Excerpta Medica; EMBiology; Scopus; Chemical Abstracts; ProQuest
The views, opinions, findings, conclusions and recommendations set forth in any Journal article are solely those of the authors of those articles and do not necessarily reflect the views, policy or position of the Journal, its Publisher, its editorial staff or any affiliated Societies and should not be attributed to any of them.
Instructions to Authors
Manuscripts must be submitted online using the following URL:
http://mc.manuscriptcentral.com/hum
Please read all the instructions to authors before submitting your article.
To help defray the cost of printing, the Publisher requests that page charges of $75 per printed page be paid by all authors who have funds available from research grants or from their institutions. It should be noted that ability to pay page charges is not a prerequisite for publication in the Journal.
Summary of Journals in the Human Gene Therapy Program
Human Gene Therapy (HGT) is our flagship journal having the richest tradition of any journal in the field of cell and gene therapy. Human Gene Therapy just celebrated its 25th anniversary and has published over 4,000 papers, reviews, and editorials. Over the last 2 years we launched two sibling journals including Human Gene Therapy Methods and Human Gene Therapy Clinical Development. In assessing your manuscript for submission to one of these three journals, please consider the following criteria.
HGT Clinical Development
This journal includes pre-clinical papers focusing on pharmacology/toxicity/bio-distribution experiments used in the review of proposed clinical trials and will contain sufficient information to allow referencing between products within the same technology platform when appropriate. If the safety studies help define novel mechanisms and/or is of very broad interest to the field, it should be considered for publication in Human Gene Therapy. Recognizing the importance of all clinical data in the formative stages of the field, HGT Clinical Development will publish clinical trials of gene and cell therapy including those which are confirmatory or negative; if the clinical trial is novel and/or reports positive data, it should be considered for publication in Human Gene Therapy.
The criteria for publishing pre-clinical safety studies and clinical trials in HGT Clinical Development will be different than in most journals. We will consider the manuscript appropriate for review if the data are collected in a way that supports the conclusions and are useful in progressing the clinical development of the product; the studies do not have to be novel or to discern mechanisms. HGT Clinical Development is an excellent venue for publishing additional topics including production and characterization data of gene and cell therapy products; selected clinical protocols which describe first-in-human applications of cell and gene therapy; and topical issues related to the commercial development of gene and cell therapy products.
Selection of Journal during the submission process
During submission, you will be asked to designate your manuscript to Human Gene Therapy or Human Gene Therapy Methods or Human Gene Therapy Clinical Development. Alternatively, you may select “Open,” in which case, the Editor-in-Chief will designate your manuscript to the appropriate journal. If the Editor-in-Chief disagrees with your journal selection but believes it is suited for one of the other two journals, the corresponding author will be notified via e-mail and will have 2 weeks to agree or decline to switch Journals. If the corresponding author declines or does not respond within 2 weeks, the manuscript will be automatically withdrawn from Manuscript Central.
Changes to Authorship
Changes to the author list are NOT permitted after submission. If it becomes necessary to add or remove an author, the corresponding author should notify the editorial office at hgt@mail.med.upenn.edu. All authors will be required to sign a document acknowledging the change.
Preparation of Manuscript
NOTE:
All submitted manuscripts will be processed through plagiarism detection software. Plagiarized manuscripts will be rejected immediately.
Articles must be submitted via upload in a word processing format, preferably in Microsoft Word. Please do not include artwork within the text document. Figures and tables should be supplied in separate files, be labeled clearly, and should be in TIFF or EPS formats. Please see the Tables and Illustrations section for further details on art submission.
Types of Manuscripts
Images in Gene and Cell Therapy
Images should tell a research story and capture the imagination as well as provide scientific information about spatial relationships and context. We invite you to submit your original striking images that illustrate and illuminate your research story and that make a timely and relevant point, emphasize a new and emerging concept, or are stunning and beautiful.
The submission format is as follows:
- Main document in Microsoft Word file format (.doc, or .docx) containing a title page (title, author name[s], author affiliation[s], corresponding author contact information), short text (250-500 words), acknowledgements, and up to 4 references; and
- One original color figure. Images must be uploaded in either .eps or .tiff format with a DPI of 300 or greater. The minimum image width is 7 inches.
All submissions will be peer reviewed and upon acceptance will be indexed in Medline. Submitted images may be considered for use on the cover of the Journal. (Cost for page charges and color printing for this section will be waived through May 1, 2015. Submissions received after that date will be subjected to the usual pricing structure of the Human Gene Therapy program.)
Research Articles - General
This format represents the majority of papers published in the HGT Journal Program. The general format is summarized below. Please note, however, that the format for pre-clinical safety studies in HGT Clinical Development is different and more structured. Instructions regarding these papers follow the summary for general papers, noted below.
Title page. Title of article, name of author(s), institutional affiliation(s), and the name (with complete address, email and phone number) to whom correspondence should be directed – there can be only one corresponding author. A short title consisting of 50 characters or less should also be included. (The title page must be part of your main text file.)
Abstract. Limit to 300 words.
Introduction
Materials and Methods. Please provide sufficient information to allow for a qualified third party to attempt to reproduce your work.
Results. We prefer that the total number of tables and figures in the printed manuscript is not greater than six (6). Feel free to include relevant supporting data in a supplementary information section.* When doing so, please refer to these data in the presentation of results that appears in the published manuscript.
Discussion. If appropriate, you may integrate the Results and Discussion into a combined section. This often allows for a more concise and understandable story.
Acknowledgments. Any contributor who does not meet the criteria for authorship should be listed in the Acknowledgments section. Examples of a contributor may be individuals who provided technical support, writing assistance, or provided research materials. All funding sources, institutional and corporate, should be listed in this section.
Disclosure Statement. Immediately following the Acknowledgments section, there should be a header entitled, Author Disclosure Statement. The corresponding author should prepare this statement and include the appropriate information for EACH author, thereby representing that competing financial interests of all authors have been appropriately disclosed according to the policy of the Journal. It is important that all conflicts of interest, whether they are actual or potential, be disclosed. If no conflicts exist, the authors must state “No competing financial interests exist.” Articles submitted without an “Author Disclosure Statement” will not be reviewed. Conflicts include:
1. Competing Interests. A competing interest exists when an individual (or the individual’s institution) has financial or personal relationships that may inappropriately influence his actions. These competing interests may be potential or actual, financial or other.
2. Personal Financial Interests. Stocks or shares in a company that may gain or lose financially from publication of the article; consulting fees or other remuneration from an organization that may gain or lose financially from publication of the article; patents or patent applications that are owned by or licensed to companies/institutions that may gain or lose value from publication of the article.
3. Funding. Research support by organizations that may gain or lose financially from publication of the article. This support includes salary, equipment, supplies, honoraria, reimbursement or prepayment for attending symposia, and other expenses.
4. Employment. Recent (within the past 5 years), current, or anticipated employment by an organization that may gain or lose financially from publication of the article.
5. Other Competing Interests. Any personal relationship which may inappropriately affect the integrity of the research reported (by an author) or the objectivity of the review of the manuscript (by a reviewer or Editor), for example, competition between investigators, previous disagreements between investigators, or bias in professional judgment.
References. (Updated and revised November 2014)
The reference list should be prepared in numerical order (not alphabetical), and in order of citation within text. References shouldbe double spaced and include the complete titles of cited articles. In-text citations should be cited in order of appearance, superscripted, and without parentheses.
If more than three authors are listed on an article, list the first three authors and use et al. (not italic) after the third author’s name.
Examples:
Journal citation: Pace N. Structure and synthesis of the ribosomal ribonucleic acid of prokaryotes. Bacteriol Rev 1973;37:562–603.
Book citation: Brosius J. Expression vectors employing A, trp, lac, and lpp-derived promoters. In: Vectors: A Survey of Molecular Cloning Vectors and Their Uses. RL Rodriguez and DT Denhardt, eds. (Butterworth Publishers, Stoneham, MA). 1988; pp. 205–225.
When references to an unpublished source are used, supply the researcher’s name and date. If work is in press, give journal in which it is to be published, and include "in press" in parenthesis.
Abbreviations of journal titles in reference section should follow the style of MEDLINE (http://www.ncbi.nlm.nih.gov/nlmcatalog).
Reviews, Commentaries and Editorials
These are usually solicited by the Editorial Board, but we welcome unsolicited submissions of this type. A pre-submission inquiry regarding an unsolicited review, commentary, or editorial should be submitted to the Editor-in-Chief at wilsonjm@mail.med.upenn.edu. If found to be of potential interest, a formal submission will be encouraged which, after submission, will be sent out for external review as well as internal review by members of the Editorial board.
Research Articles: Pre-clinical safety assessment for HGT Clinical Development
Manuscripts submitted to HGT Clinical Development describing pre-clinical safety studies should follow a format different than that of regular Research Articles as described in detail below. The organization is: Abstract, Intrduction, and a combined Results and Discussion section. Materials and Methods and any additional data should be included in a supplementary section.
Title page and Abstract. Same as for general Research Articles
Introduction. Provide sufficient context to understand the importance of the studies. Comment on the disease being treated and cell and gene therapy approaches being considered. Specifically reference and comment on published data which support the feasibility of the therapeutic approach that is the topic of the safety study.
Results and Discussion. Combine Results and Discussion and include the following sections:
1. Clinical Trial. A brief summary of the proposed clinical trial that the pre-clinical study is/was intended to support should be included in this pre-clinical paper since the reader needs context as to why the pre-clinical study is being performed. Include a description of the investigational agent being evaluated. Refer to the Materials and Methods section for more details regarding the vector/cells.
2. Objectives and Study Design. Describe the goals of the pre-clinical study followed by a detailed description of the actual design of the pre-clinical study. Feel free to use tables and/or figures as necessary to describe study design. Provide a rationale for selection of animal species/animal models and/or cell lines used as well as the basis for actual structure of the study. Include a description of the similarities/differences between the model and humans (e.g., pathophysiology, disease progression, and severity). Also comment on the timing of vector/cell administration relative to disease onset and disease progression.
3. Summary of Data. Primary data should be presented in the manuscript in summary form. A more detailed description of the data should be provided in supplementary information if it is not thoroughly covered in the printed paper (see below). An important challenge will be determining what of the source data should be included in the paper and how will it be distributed between the Results/Discussion section of the printed manuscript vs. in the supplementary section. Please keep tables and figures in the printed manuscript to no greater than six (6). Some guidance is provided below for general categories of papers.
a. Acute in vivo toxicity. Substantial quantities of data are generated in these studies not all of which should be published. The printed manuscript should describe all positive findings (demonstration of efficacy and toxicities) and pertinent negative findings (the notable absence of toxicities in target organs/tissues) at least in summary form. The printed manuscript should list all assays/test performed and note that they were within normal limits if in fact they were all normal. The supplementary information can provide more detailed information of the pertinent negative and positive findings.
b. Biodistibution. These data should be described at least in summary form in the printed manuscript; included in the printed manuscript should be details of the assay. The supplementary information can provide data for individual animals if that seems to be of interest.
c. Genotoxicity. All data related to the presence or absence of cell transformation should be included in the manuscript. Some ancillary data such as details of integration or sequence analysis can go in the supplement.
Conclusions. Please discuss the findings in the context of how they will/have influence the design of the clinical trial. Also consider the data in the context of previous work performed with similar investigational agents. This is important in providing a data base relevant to platform technologies to determine if there are common features of the platform that can help predict toxicity. Examples of platforms include bio-distribution studies with a common AAV capsid or genotoxicity studies with lentiviral vectors that share identical genome structures and envelope proteins.
Supplementary information. Feel free to include relevant supporting data in a supplementary information section that will appear online-only. When doing so, please refer to this information and data in the presentation of results that appears in the published manuscript.
1. Materials and Methods.
a. Investigational agent. Describe how the vector/cells were prepared including sufficient detail to allow for comparisons to studies using similar vectors/cells. Include all data regarding the characterization of the product.
b. Assays. Describe each assay used in the analysis of the experiment, including information about controls, assay sensitivity and/or specificity if available. Provide information regarding the state of validation of each assay such as: research based, an internal qualified assay, or a contract assay. For histopathology include details about the criteria used for assessing abnormal findings and how and who read the pathology.
c. Procedures. Details regarding non-standard procedures should be provided especially those involving in vivo work such as the conditions of administering the investigational drug.
d. Statistical methods.
2. Quality assurance. Provide information regarding steps involved in assuring the quality of the data. This can range from a study performed under full GLP to a research based study; the study does not have to be full GLP to be value to the community and relevant to the Journal. Details of the QA program should be provided. Indicate if there was no formal QA program.
3. Results. Include more detailed data for all relevant findings including positive results and pertinent negative results. The authors have some latitude as to the amount of detail that is required although it should be much more focused than a final study report but more detailed than just a summary table.
Acknowledgments, Disclosure Statement, and References. Same as for regular Research Articles
Reviews, Commentaries, and Editorials for HGT Clinical Development
These are usually solicited by the Editorial Board. However, we welcome unsolicited submissions of this type. A pre-submission inquiry regarding an unsolicited review, commentary, or editorial should be submitted to the Editor-in-Chief at wilsonjm@mail.med.upenn.edu. If found to be of potential interest, a formal submission will be encouraged which, after submission, will be sent out for external review as well as internal review by members of the Editorial Board.
Clinical Protocols for HGT Clinical Development
In selected cases, we will consider publishing clinical protocols without clinical data. Criteria for acceptance will be the novelty of the proposed trial giving priority to first in human studies. Please submit a pre-submission inquiry to the Editor-in-Chief at wilsonjm@mail.med.upenn.edu before submitting the protocol focusing on the novelty of the trial.
The Protocol should be no longer than 4500 words and should be organized as follows:
1. Introduction. Very brief background including description of disease and pre-clinical and clinical data to support the efficacy and safety of the proposed study.
2. Study Objectives
3. Study Design. including primary and secondary endpoints. Include in this section a discussion of the dose levels/dose regimens and how they were selected based on pre-clinical data or other clinical studies which use the same or a similar investigational agent.
4. Subject Selection and Withdrawal
a. Inclusion Criteria
b. Exclusion Criteria
c. Subject Recruitment and Screening
d. Informed Consent: Include an example of a consent document in Supplementary information
5. Study Drug. Brief description of the investigational agent and how it is produced
6. Study Procedures. Include a table of events. Also describe in some detail the specifics of the vector/cell infusion.
7. Statistical Plan
8. Safety and Adverse Events
a. Definitions
b. Reporting of serious adverse events and unanticipated problems
9. Risk/Benefit Assessment
LETTERS TO THE EDITOR(S):
Letters to the Editor(s) are welcomed, but with a 500-word limit and no more than one (1) table OR figure, and with a maximum of four (4) references.
Tables and Illustrations
Use Arabic numerals to number tables. Do not repeat information that is given in the text, and do not make a table for data that can be given in the text in one or two sentences. Provide titles for all tables. Define all acronyms in table footnotes. All other types of table footnotes should be designated using superscript letters, not symbols.
Please follow these guidelines for submitting figures:
- Upload each image as a separate file. Do NOT include figures and/or tables in the manuscript file.
- Do NOT embed art files into a Word or PDF document. Figures provided in this format cannot be used for production purposes.
- Line illustrations must be submitted at 900 DPI.
- Halftones and color should be submitted at a minimum of 300 dpi.
- Save as either TIFF or EPS files. JPEG files are for screen representation-quality only and will print very poorly during the printing process. To ensure proper print quality, please submit only TIFF or EPS files.
-
Color art must be saved as CMYK - not RGB. (NB: If RGB files are submitted, the files will be converted to CYMK and some color variation will occur.)
Black and white art must be submitted as grayscale – not RGB.
- Do NOT submit PowerPoint, PDF, Bitmap, or Excel as figure or table files.
- When naming your figure files, please label them with the main author’s last name, followed by the figure number. (Ex: SmithFigl; SmithTable1). Avoid using punctuation in file names if possible.
- Label figures and tables inside the files in addition to naming the file with the figure or table number. (ie: When figures or table files are opened, the figure or table number should appear inside the file.)
Additional Information About Converting Figure Files:
Converting Word or Excel files: The best and easiest way to convert Word or Excel files into a format which is suitable for print is to scan them using the below guidelines:
If you need directions on how to convert a Power Point slide to acceptable format go to: www.liebertpub.com/MEDIA/pdf/ppconvert.pdf
A legend should be supplied for each illustration, and all legends numbered consecutively and provided (double spaced) in a separate file. Do not include the figure legend as part of the figure file. All symbol definitions should be in the figure legend, not as a key within the figure. If possible please use Arial font for figure text. Figures should be numbered in the order cited in the text. Images should not show the name of the manufacturer or reveal patients’ names. Please keep in mind that most figures will need to be sized according to journal style, so please do not submit large figures/graphs that contain small type, as the text within the figure will not be readable after reduction.
The Journal will publish color photographs, but the author will be charged for the cost of color printing. For further details, contact the Publisher.
Permissions
The author must obtain permission whenever it is required in conjunction with the reproduction of material such as figures and tables from copyrighted material. Written permission must be obtained from the publisher of the journal or book concerned. The publication from which the figure or table is taken must be listed in the reference list. Finally, a footnote to a reprinted table, or of the legend of a reprinted figure should read, “Reprinted by permission from Jones et al.” and list the appropriate reference. All permissions listings must be shown in the manuscript—they cannot be entered on proofs.
Competing Interest for Reviewers
The Editors leave it to the discretion of the invited reviewer to state whether or not they have any type of conflict with any of the authors on the manuscript. Such conflicts might include recent or ongoing collaborations with the authors; having previously reviewed the manuscript in its draft stages; the reviewer is in direct competition with the authors or has a financial interest in the outcome of the manuscript. Editors will take this information into consideration when deciding whether or not to use the reviewer, or in evaluation of the reviewer’s comments. Should the reviewers feel they cannot be objective in their review for financial, personal or other reasons, they should decline to review the manuscript.
We do not exclude reviewers who previously reviewed a specific manuscript for another journal. The comments offered by a qualified reviewer are valuable to the peer-review process and it is important for the reviewer to consider that the author may have already revised the manuscript based on his/her previous comments.
Competing Interest for Editor-in-Chief and Associate Editors
The Editor-in-Chief and Associate Editors will recuse themselves from participating in the review process of any manuscript in which there is a potential or actual competing interest.
Confidentiality
Editors and Reviewers must maintain strict confidentiality of manuscripts during the peer-review process. Sharing a manuscript in whole or in part, outside the scope of what is necessary for assessment, is impermissible prior to the manuscript’s official publication date.
Sharing of Materials Policy
Authors must honor any reasonable request for materials, methods, or data necessary to reproduce or validate the research findings.
Cover Art
Authors of manuscripts accepted by the flagship journal, Human Gene Therapy, who have interesting images related to their paper, regardless of whether the images are published within the manuscript itself, are encouraged to submit them for consideration for the Journal cover. Please ensure they are not submitted or published elsewhere. Images should be high resolution—at least 300 dpi and measuring 5"×5"—.EPS or .TIFF files, and saved as CMYK, not RGB. Send images or questions to hgt@mail.med.upenn.edu.
Reprints
Reprints may be ordered by following the special instructions that will accompany the proofs, and should be ordered at the time the corresponding author returns the corrected page proofs to the Publisher. Reprints ordered after the issue is printed will be charged at a substantially higher rate.
Human Gene Therapy, HGT Methods and HGT Clinical Development will distribute or make available short press releases on newsworthy articles.
Publisher
With the addition of HGT Clinical Development in 2013, 22 issues in the Human Gene Therapy Program are published annually by Mary Ann Liebert, Inc., 140 Huguenot Street, New Rochelle, NY 10801-5215. Telephone: 914-740-2100; Fax: 914-740-2108; Email: info@liebertpub.com; Online: www.liebertpub.com
[*] Supplementary information. We encourage the judicious use of a supplementary (online-only) information section. All information/data in the supplementary information should be referred to in the printed manuscript including reference to specific tables and figures in the supplement. Upload supplementary tables, figures, and legend as separate files. The paper should be written so that the paper which appears in the printed journal contains all data which are key to the conclusions and important for the reader to have direct access to when reading the paper. Other supporting data and text are appropriate for the supplementary section.
Editorial Board
Editor-in-Chief
James M. Wilson, MD, PhD
University of Pennsylvania
Perelman School of Medicine
Gene Therapy Program
Department of Pathology and Laboratory Medicine
Suite 2000, Translational Research Laboratories
125 S. 31st Street
Philadelphia, PA 19104-3403
Ph: 215-898-0819
Fax: 215-494-5443
Email: wilsonjm@mail.med.upenn.edu
Deputy Editor
Thierry VandenDriessche, PhD
Head - Division of Gene Therapy
& Regenerative Medicine
Faculty of Medicine & Pharmacy
Free University of Brussels (VUB)
Laarbeeklaan 103
Building A
B-1090 Brussels
Belgium
Ph: 32-477-529653
Fax: 32-2-4774159
Email: thierry.vandendriessche@vub.ac.be
Human Gene Therapy Editor
Mark A. Kay, MD, PhD
Departments of Pediatrics and Genetics
Stanford University School of Medicine
269 Campus Dr. #2105
Stanford, CA 94305
Ph: 650-498-6531
Fax: 650-498-6540
Email: markay@stanford.edu
Methods Editor
Hildegard Büning, PhD
Laboratory for AAV Vector Development
Center for Molecular Medicine Cologne (CMMC)
University of Cologne
CMMC Research Building
Robert-Koch-Str. 21
50931 Cologne
Germany
Email: hildegard.buening@uk-koeln.de
Clinical Development Editor
Barry J. Byrne, MD, PhD
Powell Gene Therapy Center
University of Florida, College of Medicine
1600 SW Archer Road, RG-183
Gainesville, FL 32610
Email: bbyrne@ufl.edu
Associate Editors
Ian E. Alexander, MD, PhD
Sydney Children's Hospitals Network
and Children's Medical Research Institute
Corner Hawkesbury Rd & Hainsworth St
Locked Bag 4001
Westmead, NSW
2145 Sydney Australia
Ph: 61-2-98453031
Fax: 61-2-98451317
Email: ian.alexander@health.nsw.gov.au
Terence R. Flotte, MD
Gene Therapy Center
Departments of Pediatrics and Microbiology
& Physiologic Systems University
of Massachusetts Medical School
55 Lake Avenue North, Suite S1-340
Worcester, MA 01655
Ph: 508-856-2107
Fax: 508-856-8181
Email: terry.flotte@umassmed.edu
Yu-quan Wei, MD, PhD
National Key Laboratory of Biotherapy and
Cancer Center
West China Hospital, West China Medical
School, Sichuan University
Gaopeng Street, Keyuan Street Road 4, No. 1
Chengdu, Sichuan 610041
P.R. China
Ph: 86-28-851644059
Fax: 86-28-85164060
Email: yuquanwei@scu.edu.cn
Financial Conflicts of Interest for the Editorial Leadership of the Journal can be found by clicking here.
Scientific Editorial Board
Janet Benson, PhD, DABT
Lovelace Respiratory Research Institute
Attilio Bondanza, MD, PhD
San Raffaele Scientific Institute
Joy A. Cavagnaro, PhD, DABT, ATS
Access BIO
Jeffrey D. Chulay, MD
Applied Genetic Technologies Corporation
Thomas J. Conlon, PhD
University of Florida
Giulio Cossu , MD
University College London
Larry A. Couture, PhD
Beckman Research Institute of City of Hope
Boris Fehse, PhD
University Medical Center Hamburg-Eppendorf
Sarah Ferber, PhD
Sackler School of Medicine, Tel-Aviv University
Marie Cristina Galli, PhD
Istituto Superiore di Sanità
Gloria Gonzalez-Aseguinolaza, PhD
Foundation for Applied Medical Research (FIMA), University of Navarra
Ruben Hernandez-Alcoceba MD, PhD
Foundation for Applied Medical Research (FIMA), University of Navarra
María Lamana Luzuriaga, MD, PhD
CIEMAT/CIBERER
Francesco M. Marincola, MD
Sidra Medical and Research Center
Fulvio Mavilio, PhD
Généthon
Maritza McIntyre, PhD
REGENX Biosciences
Otto-Wilhelm Merten, PhD
Généthon
Kyriacos Mitrophanous, PhD
Oxford BioMedica (UK) Ltd.
Jose M. Moraleda MD, PhD
University of Murcia
Sarah Moyle, PhD
University of Oxford
Christian Mueller PhD, ScM
University of Massachusetts Medical School
Gopalan Narayanan, MD
NDA Regulatory Science Ltd
Hans Ovelgönne, PhD
Dutch Medicines Evaluation Board
Harald Petry, PhD
uniQure B.V.
Felipe Prosper, MD
University of Navarra
Paula Salmikangas, PhD
Finnish Medicines Agency
Christian K. Schneider, MD
Danish Health and Medicines Authority
Richard O. Snyder, PhD
University of Florida College of Medicine
Renata Stripecke, PhD
Hannover Medical School
Jean-Hugues Trouvin, PhD
Paris Descartes University
Sam Wadsworth, PhD
Dimension Therapeutics
J. Fraser Wright, PhD
University of Pennsylvania Perelman School of Medicine and
The Children’s Hospital of Philadelphia
Editorial Office
Scientific News Editors
Christian Hinderer
Sadik H. Kassim, PhD
Jennifer Joseph
Editorial Manager
Human Gene Therapy
University of Pennsylvania
125 S. 31st St., Ste. 2000 TRL
Philadelphia, PA 19104
Ph: 215-898-0819
Fax: 215-494-5443
Email: hgt@mail.med.upenn.edu
Founding Editor
James M. Wilson, MD, PhD
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Human Gene Therapy 
This journal may be purchased separately.
Human Gene Therapy Methods 
Human Gene Therapy Clinical Development 
