[HTML][HTML] Therapeutic AAV gene transfer to the nervous system: a clinical reality

E Hudry, LH Vandenberghe - Neuron, 2019 - cell.com
Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

[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 …

Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system

BL Davidson, CS Stein, JA Heth… - Proceedings of the …, 2000 - National Acad Sciences
Recombinant adeno-associated virus vectors based on serotype 2 (rAAV2) can direct
transgene expression in the central nervous system (CNS), but it is not known how other …

Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors

S Zolotukhin, M Potter, I Zolotukhin, Y Sakai, S Loiler… - Methods, 2002 - Elsevier
Recombinant adeno-associated viral (rAAV) vectors based on serotype 2 are currently being
evaluated most extensively in animals and human clinical trials. rAAV vectors constructed …

[HTML][HTML] Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain

CN Cearley, JH Wolfe - Molecular therapy, 2006 - cell.com
Recombinant adeno-associated viral (AAV) vectors can transduce cells of the CNS, resulting
in long-term expression. AAV vector transduction varies depending on the serotype used …

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 …

[HTML][HTML] Targeting T cell malignancies using CAR-based immunotherapy: challenges and potential solutions

LC Fleischer, HT Spencer, SS Raikar - Journal of hematology & oncology, 2019 - Springer
Chimeric antigen receptor (CAR) T cell therapy has been successful in treating B cell
malignancies in clinical trials; however, fewer studies have evaluated CAR T cell therapy for …

Facilitation of affiliation and pair-bond formation by vasopressin receptor gene transfer into the ventral forebrain of a monogamous vole

LJ Pitkow, CA Sharer, X Ren, TR Insel… - Journal of …, 2001 - Soc Neuroscience
Behaviors associated with monogamy, including pair-bond formation, are facilitated by the
neuropeptide vasopressin and are prevented by a vasopressin receptor [V1a receptor …

Klotho gene delivery prevents the progression of spontaneous hypertension and renal damage

Y Wang, Z Sun - Hypertension, 2009 - Am Heart Assoc
Klotho is a recently discovered antiaging gene. The objective of this study was to test the
hypothesis that klotho gene delivery attenuates the progression of spontaneous …

Genetic fate of recombinant adeno-associated virus vector genomes in muscle

BC Schnepp, KR Clark, DL Klemanski… - Journal of …, 2003 - Am Soc Microbiol
Recombinant adeno-associated virus (rAAV) vectors are promising human gene transfer
vectors, because they mediate long-term gene expression in vivo. The vector DNA form …