Results 31 to 40 of about 6,002 (83)

First-in-human study of TN-201, an AAV9 gene replacement therapy in MYBPC3-associated hypertrophic cardiomyopathy: initial safety, pharmacodynamic, and imaging results from MyPEAK-1. [PDF]

Cardiovasc Res
Desai MY, Nagueh SF, Giudicessi JR, Previs MJ, Kellner D, Pollman MJ, Varfaj F, Robertson LA, Sonicheva-Paterson N, Pushkin R, Mangal B, Tomlinson L, Harrison WG, Yamamoto L, Argast G, Lombardi LM, Ivey KN, Tingley WG.   +17 more
europepmc   +1 more source

Mosaic AAV mediates efficient transduction across the central and peripheral nervous systems. [PDF]

Mol Brain
Jiang M, Yin W, Mo X, Wang Z, Qiu M, Dai ZM.   +5 more
europepmc   +1 more source

Pervasive horizontal transfer of adeno-associated virus capsid genes. [PDF]

Proc Natl Acad Sci U S A
Gifford RJ.
europepmc   +1 more source

Compact Calm1 promoter enables AAV mediated neuron-targeted expression in human iPSC-derived brain organoids. [PDF]

Sci Rep
Klockner I, Yeturi M, Rust TE, Stein JL, Zylka MJ.   +4 more
europepmc   +1 more source

Transcriptomic Profiling Reveals Divergent Immune Responses to AAV1 and AAV-ie in Mice Inner Ear. [PDF]

Int J Med Sci
Shi D, Han L, Li C, Geng G, Jiang L, Chen X, Yang F, Feng Y, Luo J, Shu Y.   +9 more
europepmc   +1 more source

The Impact of QSP Modeling on the Design and Optimization of Gene Therapy Approaches. [PDF]

CPT Pharmacometrics Syst Pharmacol
Rayad N, Chowdhury EA, Meno-Tetang GML.
europepmc   +1 more source

Methodological validation of a cryo-TEM-based detection method for empty capsid ratio in recombinant adeno-associated virus. [PDF]

Biochem Biophys Rep
Xu L, Chen J, Zhu W, Zhao Y, Zheng F, Du R, Jiang Y, Yang Y, Chen Y, Dong Y, Zhang Z, Tong Y.   +11 more
europepmc   +1 more source

Frameless intraputaminal delivery of gene therapy with eladocagene exuparvovec in patients with aromatic L-amino acid decarboxylase deficiency: safe and efficient results. [PDF]

Childs Nerv Syst
Mai R, Reshchikov D, Popov V, Gorelikov S, Zakharova E, Mikhaylova S.   +5 more
europepmc   +1 more source

Triple rAAV9 Vector Combinations Encoding Broadly Neutralizing Antibodies Effectively Suppress HIV-1 Infection in Humanized Mice. [PDF]

Int J Mol Sci
Leontyev DS, Urusov FA, Glazkova DV, Belugin BV, Mitiushina AA, Tsyganova GM, Yudin SM, Bogoslovskaya EV, Shipulin GA.   +8 more
europepmc   +1 more source

Adeno-associated virus and hepatocytes: frenemies? [PDF]

Blood Adv
Pierce GF.
europepmc   +1 more source