Viral vectors for gene delivery to the central nervous system
TB Lentz, SJ Gray, RJ Samulski - Neurobiology of disease, 2012 - Elsevier
The potential benefits of gene therapy for neurological diseases such as Parkinson's,
Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer's are enormous. Even a delay …
Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer's are enormous. Even a delay …
[HTML][HTML] Gene therapy in animal models of autosomal dominant retinitis pigmentosa
B Rossmiller, H Mao, AS Lewin - Molecular vision, 2012 - ncbi.nlm.nih.gov
Gene therapy for dominantly inherited genetic disease is more difficult than gene-based
therapy for recessive disorders, which can be treated with gene supplementation. Treatment …
therapy for recessive disorders, which can be treated with gene supplementation. Treatment …
Gene therapy restores vision and delays degeneration in the CNGB1−/− mouse model of retinitis pigmentosa
S Koch, V Sothilingam, M Garcia Garrido… - Human molecular …, 2012 - academic.oup.com
Retinitis pigmentosa (RP) is a group of genetically heterogeneous, severe retinal diseases
commonly leading to legal blindness. Mutations in the CNGB1a subunit of the rod cyclic …
commonly leading to legal blindness. Mutations in the CNGB1a subunit of the rod cyclic …
Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer
WE Grose, KR Clark, D Griffin, V Malik, KM Shontz… - PloS one, 2012 - journals.plos.org
The dysferlinopathies comprise a group of untreatable muscle disorders including limb
girdle muscular dystrophy type 2B, Miyoshi myopathy, distal anterior compartment …
girdle muscular dystrophy type 2B, Miyoshi myopathy, distal anterior compartment …
Comparative analysis of DNA nanoparticles and AAVs for ocular gene delivery
Gene therapy is a critical tool for the treatment of monogenic retinal diseases. However, the
limited vector capacity of the current benchmark delivery strategy, adeno-associated virus …
limited vector capacity of the current benchmark delivery strategy, adeno-associated virus …
Novel mini–dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma
Y Zhang, D Duan - Human gene therapy, 2012 - liebertpub.com
Abstract Six-to 8-kb mini-dystrophin genes are promising candidates for Duchenne muscular
dystrophy (DMD) gene therapy. Several dual adeno-associated virus (AAV) mini-dystrophin …
dystrophy (DMD) gene therapy. Several dual adeno-associated virus (AAV) mini-dystrophin …
Native molecular state of adeno-associated viral vectors revealed by single-molecule sequencing
P Kapranov, L Chen, D Dederich, B Dong, J He… - Human gene …, 2012 - liebertpub.com
The single-stranded genome of adeno-associated viral (AAV) vectors is one of the key
factors leading to slow-rising but long-term transgene expression kinetics. Previous …
factors leading to slow-rising but long-term transgene expression kinetics. Previous …
Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients
P Colella, A Auricchio - Human gene therapy, 2012 - liebertpub.com
Inherited retinopathies (IRs) are common and untreatable blinding conditions inherited
mostly as monogenic due to mutations in genes expressed in retinal photoreceptors (PRs) …
mostly as monogenic due to mutations in genes expressed in retinal photoreceptors (PRs) …
Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitation
We previously reported that self-complementary adeno-associated virus (scAAV) type 2
genomes of up to 3.3 kb can be successfully encapsidated into AAV2 serotype capsids …
genomes of up to 3.3 kb can be successfully encapsidated into AAV2 serotype capsids …
Efficacy of codelivery of dual AAV2/5 vectors in the murine retina and hippocampus
A Palfi, N Chadderton, AG McKee… - Human gene …, 2012 - liebertpub.com
Recombinant adeno-associated virus (AAV) represents an efficient system for neuronal
transduction. However, a potential drawback of AAV is its restricted packaging capacity of …
transduction. However, a potential drawback of AAV is its restricted packaging capacity of …