Evaluating the state of the science for adeno-associated virus integration: an integrated perspective

DE Sabatino, FD Bushman, RJ Chandler, RG Crystal… - Molecular Therapy, 2022 - cell.com
On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …

Integration of adeno-associated virus (AAV) and recombinant AAV vectors

DM McCarty, SM Young Jr, RJ Samulski - Annu. Rev. Genet., 2004 - annualreviews.org
▪ Abstract The driving interest in adeno-associated virus (AAV) has been its potential as a
gene delivery vector. The early observation that AAV can establish a latent infection by …

A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells

GN Nguyen, JK Everett, S Kafle, AM Roche… - Nature …, 2021 - nature.com
Nine dogs with hemophilia A were treated with adeno-associated viral (AAV) gene therapy
and followed for up to 10 years. Administration of AAV8 or AAV9 vectors expressing canine …

Universal real-time PCR for the detection and quantification of adeno-associated virus serotype 2-derived inverted terminal repeat sequences

C Aurnhammer, M Haase, N Muether… - … Gene Therapy, Part B …, 2012 - liebertpub.com
Viral vectors based on various naturally occurring adeno-associated virus (AAV) serotypes
are among the most promising tools in human gene therapy. For the production of …

Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue

D Duan, P Sharma, J Yang, Y Yue, L Dudus… - Journal of …, 1998 - Am Soc Microbiol
Adeno-associated viral (AAV) vectors have demonstrated great utility for long-term gene
expression in muscle tissue. However, the mechanisms by which recombinant AAV (rAAV) …

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

H Nakai, SR Yant, TA Storm, S Fuess, L Meuse… - Journal of …, 2001 - Am Soc Microbiol
Recombinant adeno-associated virus (rAAV) vectors stably transduce hepatocytes in
experimental animals. Although the vector genomes are found both as extrachromosomes …

Adeno-associated viruses undergo substantial evolution in primates during natural infections

G Gao, MR Alvira, S Somanathan… - Proceedings of the …, 2003 - National Acad Sciences
Adeno-associated viruses (AAVs) are single-stranded DNA viruses that are endemic in
human populations without known clinical sequelae and are being evaluated as vectors for …

The role of recombinant AAV in precise genome editing

S Bijlani, KM Pang, V Sivanandam, A Singh… - Frontiers in Genome …, 2022 - frontiersin.org
The replication-defective, non-pathogenic, nearly ubiquitous single-stranded adeno-
associated viruses (AAVs) have gained importance since their discovery about 50 years …

[HTML][HTML] Adeno-associated virus vector integration

DR Deyle, DW Russell - Current opinion in molecular therapeutics, 2009 - ncbi.nlm.nih.gov
Adeno-associated virus (AAV) vectors efficiently transduce various cell types and can
produce long-term expression of transgenes in vivo. Although AAV vector genomes can …

Gene therapy: promises and problems

A Pfeifer, IM Verma - Annual review of genomics and human …, 2001 - annualreviews.org
Gene therapy can be broadly defined as the transfer of genetic material to cure a disease or
at least to improve the clinical status of a patient. One of the basic concepts of gene therapy …