Cited by 205Virus-based gene therapy researchViral Infectious Diseases and Gene Expression in InsectsRNA Interference and Gene Delivery

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Manufacturing and characterizing AAV-based vectors for use in clinical studies

Wright, J F·2008·Gene Therapy

DOI 10.1038/gt.2008.65

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Abstract

Recombinant adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great promise for human gene therapy. A major challenge for translation of promising research to clinical development is the manufacture and certification of AAV vectors for clinical use. This review summarizes relevant aspects of current Good Manufacturing Practice, focusing on considerations and challenges specific for recombinant AAV.

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Authors: Wright, J. F.
Affiliations: Children's Hospital of Philadelphia (US)
Corresponding author: Wright, J F
ORCID: Wright, J F
Venue: Gene Therapy
Publisher: Springer Science and Business Media LLC
Published: 2008-04-17
Volume: 15
Issue: 11
Pages: 840-848
ISSN: 0969-7128 (ISSN-L)
1476-5462
DOI: 10.1038/gt.2008.65
OpenAlex: W2048382073
Cited by: 205
References: 50
Citations by year: 2026: 12 · 2025: 15 · 2024: 25 · 2023: 18 · 2022: 13 · 2021: 26 · 2020: 12 · 2019: 12 · 2018: 9 · 2017: 14 · 2016: 7 · 2015: 6 · 2014: 4 · 2013: 6 · 2012: 7
FWCI: 0.59
Type: journal article
Language: en
Open access: closed
Licence: www.springer.com/tdm (tdm)
www.springer.com/tdm (vor)
Locations: Gene Therapy (publishedVersion)
PubMed (publishedVersion)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Viral Infectious Diseases and Gene Expression in Insects
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Keywords: recombinant adeno-associated virus vectors, current good manufacturing practice, clinical translation, gene therapy vectors
MeSH terms: Dependovirus, Genetic Engineering, Genetic Vectors, Humans, Industry, Quality Control, Recombinant Proteins, Genetic Therapy

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