Next Article in Journal
Current In Vitro Models to Study Varicella Zoster Virus Latency and Reactivation
Previous Article in Journal
Orthopoxvirus Seroprevalence in Cats and Veterinary Personnel in North-Eastern Italy in 2011
Article Menu
Issue 2 (February) cover image

Export Article

Open AccessReview
Viruses 2019, 11(2), 102;

Adeno-Associated Virus (AAV) Versus Immune Response

Senior Director of Capsid Development, Rare Disease Research Unit, Pfizer Inc., 7030 Kit Creek Road, Morrisville, NC 27560, USA
Department of Genetics, Harvard Medical School, Boston, MA 02115, USA
Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA 02115, USA
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)
Full-Text   |   PDF [827 KB, uploaded 30 January 2019]   |  


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

Figure 1

This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).

Share & Cite This Article

MDPI and ACS Style

Rabinowitz, J.; Chan, Y.K.; Samulski, R.J. Adeno-Associated Virus (AAV) Versus Immune Response. Viruses 2019, 11, 102.

Show more citation formats Show less citations formats

Note that from the first issue of 2016, MDPI journals use article numbers instead of page numbers. See further details here.

Related Articles

Article Metrics

Article Access Statistics



[Return to top]
Viruses EISSN 1999-4915 Published by MDPI AG, Basel, Switzerland RSS E-Mail Table of Contents Alert
Back to Top