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 …
therapy in humans. The recent successes seen in Phase I/II clinical trials have also …
Adeno-associated virus vectorology, manufacturing, and clinical applications
JC Grieger, RJ Samulski - Methods in enzymology, 2012 - Elsevier
Adeno-associated virus (AAV) has emerged as an attractive vector for gene therapy. The
benefits of using AAV for gene therapy include long-term gene expression, the inability to …
benefits of using AAV for gene therapy include long-term gene expression, the inability to …
Intracellular transport of recombinant adeno-associated virus vectors
M Nonnenmacher, T Weber - Gene therapy, 2012 - nature.com
Recombinant adeno-associated viral vectors (rAAVs) have been widely used for gene
delivery in animal models, and are currently evaluated for human gene therapy after …
delivery in animal models, and are currently evaluated for human gene therapy after …
AAV capsid structure and cell interactions
M Agbandje-McKenna, J Kleinschmidt - Adeno-associated virus: methods …, 2011 - Springer
Abstract The Adeno-associated viruses (AAVs) are not associated with any diseases, and
their ability to package non-genomic DNA and to transduce different cell/tissue populations …
their ability to package non-genomic DNA and to transduce different cell/tissue populations …
Adeno-associated virus (AAV) versus immune response
J Rabinowitz, YK Chan, RJ Samulski - Viruses, 2019 - mdpi.com
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 …
tissue targeting, delivery across the blood–brain barrier (BBB), and host immune responses …
[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 …
PTK) signaling negatively affects intracellular trafficking and transduction efficiency of …
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells
AT Martino, E Basner-Tschakarjan… - Blood, The Journal …, 2013 - ashpublications.org
Recent clinical trials have shown that evasion of CD8+ T-cell responses against viral capsid
is critical for successful liver-directed gene therapy with adeno-associated viral (AAV) …
is critical for successful liver-directed gene therapy with adeno-associated viral (AAV) …
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors
GC Pien, E Basner-Tschakarjan, DJ Hui… - The Journal of …, 2009 - Am Soc Clin Investig
Adeno-associated virus (AAV) vectors are effective gene delivery vehicles mediating long-
lasting transgene expression. Data from a clinical trial of AAV2-mediated hepatic transfer of …
lasting transgene expression. Data from a clinical trial of AAV2-mediated hepatic transfer of …
Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8+ T cells
GL Rogers, JL Shirley, I Zolotukhin… - Blood, The Journal …, 2017 - ashpublications.org
Adeno-associated virus (AAV) is a replication-deficient parvovirus that is extensively used as
a gene therapy vector. CD8+ T-cell responses against the AAV capsid protein can, however …
a gene therapy vector. CD8+ T-cell responses against the AAV capsid protein can, however …
Engineering the AAV capsid to optimize vector–host-interactions
Highlights•Engineering of AAV capsids optimizes efficiency and/or specificity of gene
transfer.•High throughput-selection of AAV libraries identifies AAV variants with novel …
transfer.•High throughput-selection of AAV libraries identifies AAV variants with novel …