[HTML][HTML] 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 …
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …
Recombinant adeno-associated viral integration and genotoxicity: insights from animal models
RJ Chandler, MS Sands, CP Venditti - Human gene therapy, 2017 - liebertpub.com
Currently, clinical gene therapy is experiencing a renaissance, with new products for clinical
use approved in Europe and clinical trials for multiple diseases reporting positive results …
use approved in Europe and clinical trials for multiple diseases reporting positive results …
[HTML][HTML] Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to …
C Burger, OS Gorbatyuk, MJ Velardo, CS Peden… - Molecular Therapy, 2004 - cell.com
Recombinant adeno-associated virus 2 (rAAV2) has been shown to deliver genes to
neurons effectively in the brain, retina, and spinal cord. The characterization of new AAV …
neurons effectively in the brain, retina, and spinal cord. The characterization of new AAV …
[HTML][HTML] Adeno-associated virus (AAV)-mediated gene therapy for Duchenne muscular dystrophy: the issue of transgene persistence
A Manini, E Abati, A Nuredini, S Corti… - Frontiers in …, 2022 - frontiersin.org
Duchenne muscular dystrophy (DMD) is an X-linked recessive, infancy-onset
neuromuscular disorder characterized by progressive muscle weakness and atrophy …
neuromuscular disorder characterized by progressive muscle weakness and atrophy …
[HTML][HTML] Gene therapy for lysosomal storage diseases
MS Sands, BL Davidson - Molecular Therapy, 2006 - cell.com
Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders
with complex clinical phenotypes that include both systemic and central nervous system …
with complex clinical phenotypes that include both systemic and central nervous system …
Towards a neuroprotective gene therapy for Parkinson's disease: use of adenovirus, AAV and lentivirus vectors for gene transfer of GDNF to the nigrostriatal system in …
A Björklund, D Kirik, C Rosenblad, B Georgievska… - Brain research, 2000 - Elsevier
During the last few years, recombinant viral vectors derived from adenovirus (Ad), adeno-
associated virus (AAV) or lentivirus (LV) have been developed into highly effective vehicles …
associated virus (AAV) or lentivirus (LV) have been developed into highly effective vehicles …
Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway
AM Davidoff, CYC Ng, J Zhou, Y Spence, AC Nathwani - Blood, 2003 - ashpublications.org
A systematic evaluation of the influence of sex on transduction by recombinant adeno-
associated viral vector (rAAV) indicated that transgene expression after liver-targeted …
associated viral vector (rAAV) indicated that transgene expression after liver-targeted …
Dopaminergic Cell Loss Induced by Human A30P α-Synuclein Gene Transfer to the Rat Substantia Nigra
RL Klein, MA King, ME Hamby, EM Meyer - Human gene therapy, 2002 - liebertpub.com
Somatic cell gene transfer was used to express a mutant form of α-synuclein (α-syn) that is
associated with Parkinson's disease (PD) in the rat substantia nigra (SN), a brain region that …
associated with Parkinson's disease (PD) in the rat substantia nigra (SN), a brain region that …
Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2
D Sondhi, SM Kaminsky, NR Hackett… - Science translational …, 2020 - science.org
Late infantile Batten disease (CLN2 disease) is an autosomal recessive, neurodegenerative
lysosomal storage disease caused by mutations in the CLN2 gene encoding tripeptidyl …
lysosomal storage disease caused by mutations in the CLN2 gene encoding tripeptidyl …
Dose and promoter effects of adeno-associated viral vector for green fluorescent protein expression in the rat brain
RL Klein, ME Hamby, Y Gong, AC Hirko, S Wang… - Experimental …, 2002 - Elsevier
Previous studies demonstrated that the rat neuron-specific enolase (NSE) promoter is
effective for transgene expression in the brain in a variety of adeno-associated virus-2 …
effective for transgene expression in the brain in a variety of adeno-associated virus-2 …