Results 61 to 70 of about 6,270 (121)

Advances in AAV capsid engineering: Integrating rational design, directed evolution and machine learning. [PDF]

Mol Ther
Nisanov AM, Rivera de Jesús JA, Schaffer DV.   +2 more
europepmc   +1 more source

CRISPR-Cpf1-Mediated Gene-Editing System Based on a Single Bidirectional Promoter. [PDF]

Int J Mol Sci
Kim S, Kim GN, Jeong YJ, Cho J, Jang M, Hong J, Sung YH.   +6 more
europepmc   +1 more source

Treatment With Valoctocogene Roxaparvovec in a Patient With Severe Hemophilia A Led to Sustained Normal FVIII Levels. [PDF]

Haemophilia
Herbst K, Pezeshkpoor B, Klein C, Albert T, Lutz P, Strassburg C, Spengler U, Goldmann G, Oldenburg J.   +8 more
europepmc   +1 more source

The calcium awakens: new insights in cardiac gene therapy. [PDF]

Front Endocrinol (Lausanne)
Santulli G.
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

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

A New <i>Wnt1</i> Mutant Rat Model of Osteogenesis Imperfecta and Its Application in AAV9-Mediated Gene Therapy. [PDF]

Hum Mutat
Li S, Chen X, Cao Y, Han M, Guan F, Ren X, Mi H, Yang T, Li M, Zhao X.   +9 more
europepmc   +1 more source

A Rationally Designed AAV9-DM Capsid with Minimal Liver Tropism. [PDF]

Cells
Nabakowski ZC, Jaramillo IC, Tanachaiwiwat P, Keeler GD.   +3 more
europepmc   +1 more source

Classification and quantification of full vs. empty adeno-associated virus reference capsids via infrared attenuated total reflection spectroscopy. [PDF]

Anal Bioanal Chem
Lehner S, Stach R, Nilson R, Kochanek S, Kritzinger A, Sobek H, Kokoric V, Mizaikoff B.   +7 more
europepmc   +1 more source