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Immune responses to AAV vectors: overcoming barriers to successful gene therapy.
Mingozzi F, High KA. Mingozzi F, et al. Blood. 2013 Jul 4;122(1):23-36. doi: 10.1182/blood-2013-01-306647. Epub 2013 Apr 17. Blood. 2013. PMID: 23596044 Free PMC article. Review.
Gene therapy products for the treatment of genetic diseases are currently in clinical trials, and one of these, an adeno-associated viral (AAV) product, has recently been licensed. ...Small-scale clinical studies have enabled investigators to dissect t
Gene therapy products for the treatment of genetic diseases are currently in clinical trials, and one of these, an aden
Overcoming innate immune barriers that impede AAV gene therapy vectors.
Muhuri M, Maeda Y, Ma H, Ram S, Fitzgerald KA, Tai PW, Gao G. Muhuri M, et al. J Clin Invest. 2021 Jan 4;131(1):e143780. doi: 10.1172/JCI143780. J Clin Invest. 2021. PMID: 33393506 Free article. Review.
Adeno-associated virus (AAV) vectors have emerged as promising and attractive tools for in vivo gene therapy. Despite the recent clinical successes achieved with recombinant AAVs (rAAVs) for therapeutics, host immune responses agai …
Adeno-associated virus (AAV) vectors have emerged as promising and attractive tools for in vivo gene therapy. De …
Mapping an Adeno-associated Virus 9-Specific Neutralizing Epitope To Develop Next-Generation Gene Delivery Vectors.
Giles AR, Govindasamy L, Somanathan S, Wilson JM. Giles AR, et al. J Virol. 2018 Sep 26;92(20):e01011-18. doi: 10.1128/JVI.01011-18. Print 2018 Oct 15. J Virol. 2018. PMID: 30089698 Free PMC article.
Recent clinical trials have demonstrated the potential of adeno-associated virus (AAV)-based vectors for treating rare diseases. However, significant barriers remain for the translation of these vectors into widely available therapies. ...Taken togethe …
Recent clinical trials have demonstrated the potential of adeno-associated virus (AAV)-based vectors for treating rare disease …