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Human Gene Therapy

Editor-in-Chief: James M. Wilson, MD, PhD
Deputy Editor: George Dickson, BSc, PhD
Human Gene Therapy Editor: Mark A. Kay, MD, PhD
Methods Editor: Thierry VandenDriessche, 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 4.019

*2012 Journal Citation Reports® published by Thomson Reuters, 2013

Manuscript Submission

Manuscripts must be submitted online using the following URL:


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.

Human Gene Therapy

The flagship journal will consider papers of very high quality that broadly fall under the scope of cell and gene therapy. Some general categories include: studies evaluating novel approaches of cell and gene therapy in pre-clinical and clinical models; development of novel technologies; studies that help define mechanisms of action of cell and gene therapies; host interactions to vector and transplanted cells; and high-impact new methods and clinical trials. The Editors may quickly reject a manuscript soon after submission without external review if it is felt to fall outside the scope of the Journal or would not likely meet our high standards required for publication. The authors may also be given the option of re-submitting the manuscript to one of the sibling journals as described below.

HGT Methods

We encourage submission of manuscripts to HGT Methods that describe key technological advances. These manuscripts can cover a wide range of topics from techniques for detecting transgene expression, to improved vectors to better manufacturing methods and product testing assays. If the method is of very broad interest and holds the potential to substantially progress the field, it should be directed to Human Gene Therapy by the submitting author. The Editor may make this determination as well.

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


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 in Gene and Cell Therapy 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 image(s) that illustrate and illuminate your research story and that make a timely and relevant point, emphasize a new and emerging concept, and/or are stunning and beautiful.  These submissions should be uploaded under the  Manuscript Type “Images in Gene Cell Therapy,” and the Journal Option “Open” should be selected.  After a preliminary review, the Editor-in-Chief will determine for which part of the Human Gene Therapy program the submission is best suited.
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 July 2014. 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.


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. All references in text should be cited alphabetically, within year, by authors and dates. e.g. The ribosomal RNA (rrn) operons in E. coli contain single copies of each of the 16S, 23S, and 5S r(RNA) genes (Pace, 1973; Rogers and Smith, 1973; Andrews et al., 1978).


If more than two authors are involved, use et al. after the first author’s name.

e.g. (Sogin et al., 1971).


If several articles by the same authors are cited, use a lower case letter designation to indicate the individual articles.

e.g. (Dunn and Studier, 1973a,b). Use same designation in reference list.


All references in the reference section should be double spaced and listed in alphabetical order at the end of the article. References should include complete titles of cited articles.


Journal citation: Pace N. (1973). Structure and synthesis of the ribosomal ribonucleic acid of prokaryotes. Bacteriol. Rev. 37, 562–603.


Book citation: Brosius J. (1987). 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) pp. 205–225.


When dates from an unpublished source are given, supply the researcher’s name. If work is in press, give journal in which it is to be published or name of publisher. Abbreviations of journal names should follow the style of MEDLINE.


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


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:


  • All files should be scanned at 100% size

  • 300 dpi
  • Final color mode: CMYK

  •  Save file as: .TIF or .EPS

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.




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.




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 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.




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.

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.

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