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 …

Gene therapy using adeno-associated virus vectors

S Daya, KI Berns - Clinical microbiology reviews, 2008 - Am Soc Microbiol
The unique life cycle of adeno-associated virus (AAV) and its ability to infect both
nondividing and dividing cells with persistent expression have made it an attractive vector …

[HTML][HTML] Analysis of AAV serotypes 1–9 mediated gene expression and tropism in mice after systemic injection

C Zincarelli, S Soltys, G Rengo, JE Rabinowitz - Molecular therapy, 2008 - cell.com
This study examines transgene expression and biodistribution of adeno-associated virus
(AAV) pseudotyped 1–9 after tail vein (TV) injection in male mice. Using a cytomegalovirus …

[HTML][HTML] 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 …

Virus-mediated gene delivery for human gene therapy

M Giacca, S Zacchigna - Journal of controlled release, 2012 - Elsevier
After over 20years from the first application of gene transfer in humans, gene therapy is now
a mature discipline, which has progressively overcome several of the hurdles that prevented …

[HTML][HTML] A viral phospholipase A2 is required for parvovirus infectivity

Z Zádori, J Szelei, MC Lacoste, Y Li, S Gariépy… - Developmental cell, 2001 - cell.com
Sequence analysis revealed phospholipase A 2 (PLA 2) motifs in capsid proteins of
parvoviruses. Although PLA 2 activity is not known to exist in viruses, putative PLA 2 s from …

Dynamin and its role in membrane fission

JE Hinshaw - Annual review of cell and developmental biology, 2000 - annualreviews.org
▪ Abstract Dynamin, a 100-kDa GTPase, is an essential component of vesicle formation in
receptor-mediated endocytosis, synaptic vesicle recycling, caveolae internalization, and …

Dissecting virus entry via endocytosis

SB Sieczkarski, GR Whittaker - Journal of General …, 2002 - microbiologyresearch.org
Numerous virus families utilize endocytosis to infect host cells, mediating virus
internalization as well as trafficking to the site of replication. Recent research has …

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 …

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 …