Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses

L Zhong, B Li, CS Mah… - Proceedings of the …, 2008 - National Acad Sciences
Recombinant adeno-associated virus 2 (AAV2) vectors are in use in several Phase I/II
clinical trials, but relatively large vector doses are needed to achieve therapeutic benefits …

[HTML][HTML] Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression

LI Zhong, B Li, G Jayandharan, CS Mah… - Virology, 2008 - Elsevier
We have documented that epidermal growth factor receptor protein tyrosine kinase (EGFR-
PTK) signaling negatively affects intracellular trafficking and transduction efficiency of …

Basic biology of adeno-associated virus (AAV) vectors used in gene therapy

B Balakrishnan, GR Jayandharan - Current gene therapy, 2014 - ingentaconnect.com
Adeno-associated virus (AAV) based vectors have emerged as important tools for gene
therapy in humans. The recent successes seen in Phase I/II clinical trials have also …

Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors

TR Flotte - Gene therapy, 2004 - nature.com
Recombinant adeno-associated virus (rAAV) vectors are unique among the vector classes
currently available for human gene therapy in that they are based upon a class of viruses …

Insertional mutagenesis of the adeno-associated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors targeted to alternative cell-surface receptors

W Shi, GS Arnold, JS Bartlett - Human gene therapy, 2001 - liebertpub.com
Recombinant adeno-associated virus (AAV) vectors are of interest in the context of gene
therapy because of their ability to mediate efficient transfer and stable expression of …

Adeno-associated virus vector as a platform for gene therapy delivery

D Wang, PWL Tai, G Gao - Nature reviews Drug discovery, 2019 - nature.com
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 …

Small but increasingly mighty: latest advances in AAV vector research, design, and evolution

D Grimm, H Büning - Human gene therapy, 2017 - liebertpub.com
Recombinant gene delivery vectors derived from naturally occurring or genetically
engineered adeno-associated viruses (AAV) have taken center stage in human gene …

Adeno-associated virus serotypes: vector toolkit for human gene therapy

Z Wu, A Asokan, RJ Samulski - Molecular therapy, 2006 - cell.com
Recombinant adeno-associated viral (AAV) vectors have rapidly advanced to the forefront of
gene therapy in the past decade. The exponential progress of AAV-based vectors has been …

Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions

JM Riyad, T Weber - Gene therapy, 2021 - nature.com
In the last two decades, recombinant adeno-associated virus has emerged as the most
popular gene therapy vector. Recently AAV gene therapy has been approved by the FDA for …

Can adeno-associated viral vectors deliver effectively large genes?

P Tornabene, I Trapani - Human gene therapy, 2020 - liebertpub.com
Gene therapy with adeno-associated viral (AAV) vectors has reached the clinical stage for
many inherited and acquired diseases. However, due to a cargo capacity limited to< 5 kb …