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Biomedicines
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Adeno-Associated Virus Vectors for Gene Therapy
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Adeno-Associated Virus Vectors for Gene Therapy

A special issue of Biomedicines (ISSN 2227-9059). This special issue belongs to the section "Gene and Cell Therapy".

Deadline for manuscript submissions: closed (31 October 2022) | Viewed by 6666

Special Issue Editor

Dr. Ilayaraja Muthuramu

E-Mail Website
Guest Editor
Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
Interests: AAV vectors discovery research; genome editing; caspid engineering

Special Issue Information

Dear Colleagues,

We have come a long way from the newly approved FDA drugs based on the AAV vectors from ‘Bench to Bedside.’ AAV vectors are considered a valuable clinical tool resulting in the increased number of human gene therapy clinical trials at an unprecedented rate. Despite the promising results in clinical trials, Adeno (associated) viruses and vectors face challenges and major hurdles in immune responses, NAbs, manufacturing at commercial scales, and lower transduction efficiency in humans than in animal models, which all still remain significant obstacles that has to be addressed. The genetic modification of AAV and vector engineering has the potential to overcome these major hurdles. This Special Issue will highlight recent advances in novel Adeno (associated) viral vectors; a better understanding of the interaction of the virus capsid with the components of the immune system guides the development of next-generation vectors.

This Special Issue of Biomedicine will be dedicated to the following themes: current advancement in AAV viral vectors development in manufacturing, capsid engineering, immune evasion, and usage of AAV vectors in genome editing. This Special Issue will publish reviews and original research containing experimental data on AAV gene therapy. The Special Issue also includes manuscripts on technological advances of AAV gene therapy. Authors are encouraged to provide their assessment and vision for the future development of AAV vectors.

Dr. Ilayaraja Muthuramu
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Biomedicines is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • AAV Vectors
  • clinical trials
  • immune response
  • capsid engineering

Published Papers (2 papers)

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Research

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16 pages, 2931 KiB  
Article
Multiplexing AAV Serotype-Specific Neutralizing Antibodies in Preclinical Animal Models and Humans
by Hisae Kuoch, Karina Krotova, Melanie L. Graham, Mark L. Brantly and George Aslanidi
Biomedicines 2023, 11(2), 523; https://doi.org/10.3390/biomedicines11020523 - 11 Feb 2023
Cited by 2 | Viewed by 2728
Abstract
The accurate assessment of AAV-specific pre-existing humoral immunity due to natural viral infection is critical for the efficient use of clinical gene therapy. The method described in the present study applies equivalent infection conditions to each AAV serotype (AAV1, AAV2, AAV3, AAV5, AAV6, [...] Read more.
The accurate assessment of AAV-specific pre-existing humoral immunity due to natural viral infection is critical for the efficient use of clinical gene therapy. The method described in the present study applies equivalent infection conditions to each AAV serotype (AAV1, AAV2, AAV3, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, and AAVAnc80L65). In the current study, we validated the assay by assessing AAV-neutralizing antibody titers in a limited cohort of random human donors and well-established preclinical large animal models, including dogs and non-human primates (NHPs). We achieved a rapid and accurate evaluation of neutralizing titers for each individual subject that can be used for clinical enrollment based on specific AAV serotypes and individualized selection of the most suitable AAV serotype for each specific patient. Full article
(This article belongs to the Special Issue Adeno-Associated Virus Vectors for Gene Therapy)
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Review

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21 pages, 1314 KiB  
Review
Adeno-Associated Viruses for Modeling Neurological Diseases in Animals: Achievements and Prospects
by Evgenii Lunev, Anna Karan, Tatiana Egorova and Maryana Bardina
Biomedicines 2022, 10(5), 1140; https://doi.org/10.3390/biomedicines10051140 - 15 May 2022
Cited by 5 | Viewed by 3458
Abstract
Adeno-associated virus (AAV) vectors have become an attractive tool for efficient gene transfer into animal tissues. Extensively studied as the vehicles for therapeutic constructs in gene therapy, AAVs are also applied for creating animal models of human genetic disorders. Neurological disorders are challenging [...] Read more.
Adeno-associated virus (AAV) vectors have become an attractive tool for efficient gene transfer into animal tissues. Extensively studied as the vehicles for therapeutic constructs in gene therapy, AAVs are also applied for creating animal models of human genetic disorders. Neurological disorders are challenging to model in laboratory animals by transgenesis or genome editing, at least partially due to the embryonic lethality and the timing of the disease onset. Therefore, gene transfer with AAV vectors provides a more flexible option for simulating genetic neurological disorders. Indeed, the design of the AAV expression construct allows the reproduction of various disease-causing mutations, and also drives neuron-specific expression. The natural and newly created AAV serotypes combined with various delivery routes enable differentially targeting neuronal cell types and brain areas in vivo. Moreover, the same viral vector can be used to reproduce the main features of the disorder in mice, rats, and large laboratory animals such as non-human primates. The current review demonstrates the general principles for the development and use of AAVs in modeling neurological diseases. The latest achievements in AAV-mediated modeling of the common (e.g., Alzheimer’s disease, Parkinson’s disease, ataxias, etc.) and ultra-rare disorders affecting the central nervous system are described. The use of AAVs to create multiple animal models of neurological disorders opens opportunities for studying their mechanisms, understanding the main pathological features, and testing therapeutic approaches. Full article
(This article belongs to the Special Issue Adeno-Associated Virus Vectors for Gene Therapy)
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