Results 51 to 60 of about 396,665 (375)

Gene Transfer in Rodent Nervous Tissue Following Hindlimb Intramuscular Delivery of Recombinant Adeno-Associated Virus Serotypes AAV2/6, AAV2/8, and AAV2/9

Neuroscience Insights, 2019
Recombinant adeno-associated virus (rAAV) vectors have emerged as the safe vehicles of choice for long-term gene transfer in mammalian nervous system. Recombinant adeno-associated virus–mediated localized gene transfer in adult nervous system following ...
Asad Jan, Mette Richner, Christian B Vægter, Jens R Nyengaard, Poul H Jensen   +4 more
doaj   +1 more source

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. [PDF]

, 2003
A systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice ...
Davidoff, AM, Nathwani, AC, Ng, CYC, Spence, Y, Zhou, J   +4 more
core   +1 more source

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

Scientific Reports, 2020
Neutralizing antibodies directed against adeno-associated virus (AAV) are commonly found in humans. In seropositive subjects, vector administration is not feasible as antibodies neutralize AAV vectors even at low titers.
B. Bertin, P. Véron, C. Leborgne, J. Deschamps, S. Moullec, Y. Fromes, F. Collaud, S. Boutin, V. Latournerie, L. van Wittenberghe, B. Delache, R. Le Grand, N. Dereuddre-Bosquet, O. Benveniste, P. Moullier, C. Masurier, O. Merten, F. Mingozzi   +17 more
semanticscholar   +1 more source

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]

, 2001
Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A, Hanawa, H, Nathwani, AC, Nienhuis, AW, Vanin, EF, Zhou, JF   +5 more
core   +1 more source

Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors

Molecular Therapy: Methods & Clinical Development, 2022
Gene therapy using neurotropic adeno-associated virus vectors represents an emerging solution for genetic disorders affecting the central nervous system. The first approved central nervous system-targeting adeno-associated virus gene therapy, Zolgensma®,
Makoto Horiuchi, Christian J. Hinderer, Jenny A. Greig, Cecilia Dyer, Elizabeth L. Buza, Peter Bell, Jessica A. Chichester, Peter M. Hayashi, Hanying Yan, Tamara Goode, James M. Wilson   +10 more
doaj  

Adeno-associated virus as a delivery vector for gene therapy of human diseases

Signal Transduction and Targeted Therapy
Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues.
Jiang-Hui Wang, Dominic J Gessler, Wei Zhan, Thomas Gallagher, Guangping Gao   +4 more
semanticscholar   +1 more source

Adeno associated virus serotype.

Human Gene Therapy, 2020
Adeno-associated viral vectors have been successfully used in laboratory and clinical settings for efficient gene delivery. In these vectors, 96% of the AAV genome is replaced with a gene cassette of interest, leaving only the 145 bp inverted terminal ...
L. F. Earley, Laura M. Conatser, Victoria M. Lue, A. Dobbins, Chengwen Li, M. Hirsch, R. Samulski   +6 more
semanticscholar   +1 more source

Biosafety of Recombinant Adeno-associated Virus Vectors [PDF]

Current Gene Therapy, 2014
It is hoped that the use of gene transfer technology to treat both monogenetic and acquired diseases may soon become a common therapy option in medicine. For gene therapy to achieve this objective, any gene delivery method will have to meet several criteria, including ease of manufacturing, efficient gene transfer to target tissue, long-term gene ...
Dismuke, D.J., Tenenbaum, L., Samulski, R.J.   +2 more
openaire   +3 more sources

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus. [PDF]

, 2013
Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified before the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness.
Boye, S, Boye, SE, Chiodo, V, Dyka, F, Fofo, H, Hauswirth, WW, Lopes, VS, Louie, CM, Williams, DS   +8 more
core   +1 more source

LDLR-Gene therapy for familial hypercholesterolaemia: Problems, progress, and perspectives [PDF]

, 2010
Coronary artery diseases (CAD) inflict a heavy economical and social burden on most populations and contribute significantly to their morbidity and mortality rates.
Al-Allaf, FA, Coutelle, C, David, A, Harbottle, R, Themis, M, Waddington, SN   +5 more
core   +3 more sources