Adeno-associated virus vector as a platform for gene therapy delivery
By Dan Wang, P. Tai, G. Gao. Nature reviews. Drug discovery, 2019
2,093 citations recorded by the source corpus.
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to the growth of the gene therapy field. Preclinical and clinical successes in AAV-mediated gene replacement, gene silencing and gene editing have helped AAV gain popularity as the ideal therapeutic vector, with two AAV-based therapeutics gaining regulatory approval in Europe or the United States. Continued study of AAV biology and increased understanding of the associated therapeutic challenges and limitations will build the foundation for future clinical success. Adeno-associated virus (AAV) vectors represent the leading platform for therapeutic gene delivery, with two recombinant AAV gene therapy products having gained regulatory approval in Europe or the United States. Here, Gao and colleagues discuss the fundamentals of AAV and vectorology, focusing on current therapeutic strategies, clinical progress and ongoing challenges.
Subfields: Cardiology and Cardiovascular Medicine, Genetics, Molecular Biology
View Paper: https://www.semanticscholar.org/paper/4a1256ea41f3e62e64919cfcd4002bf696d02dd7
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In Cardiology and Cardiovascular Medicine
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Citations in the corpus
- Adeno-associated virus as a delivery vector for gene therapy of human diseases (2024, methodology): “16 This has advanced to successful rAAV-based clinical trials, particularly for monogenic rare diseases, resulting in regulatory approvals of rAAV gene therapy products.”