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Viruses 2019, 11(2), 102; https://doi.org/10.3390/v11020102

Adeno-Associated Virus (AAV) Versus Immune Response

1
Senior Director of Capsid Development, Rare Disease Research Unit, Pfizer Inc., 7030 Kit Creek Road, Morrisville, NC 27560, USA
2
Department of Genetics, Harvard Medical School, Boston, MA 02115, USA
3
Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA 02115, USA
4
Department of Pharmacology, School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA
*
Authors to whom correspondence should be addressed.
Received: 5 December 2018 / Revised: 9 January 2019 / Accepted: 16 January 2019 / Published: 25 January 2019
(This article belongs to the Section Animal Viruses)
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Abstract

Decades ago, Friedmann and Roblin postulated several barriers to gene therapy, including tissue targeting, delivery across the blood–brain barrier (BBB), and host immune responses. These issues remain pertinent till today. Since then, several advances have been made in elucidating structures of adeno-associated virus (AAV) serotypes, antibody epitopes, and ways to modify antibody-binding sites. AAVs capsid has also been engineered to re-direct tissue tropism, reduce ubiquitination, and promote passage across the BBB. Furthermore, the use of high(er) dose recombinant AAV (rAAV) has been accompanied by a better understanding of immune responses in both experimental animals and early clinical trials, and novel work is being performed to modulate the immune response. While the immune responses to rAAV remains a major challenge in translating experimental drugs to approved medicine, and will likely require more than a single solution, we now better understand the hurdles to formulate and test experimental solutions to surmount them. View Full-Text
Keywords: rAAV; immune response; rare diseases rAAV; immune response; rare diseases
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Rabinowitz, J.; Chan, Y.K.; Samulski, R.J. Adeno-Associated Virus (AAV) Versus Immune Response. Viruses 2019, 11, 102.

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