Results 81 to 90 of about 6,474 (125)

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

Front Endocrinol (Lausanne)
Santulli G.
europepmc   +1 more source

Reactive species generated by a cold atmospheric plasma inactivate AAV through oxidative and nitrosative stress. [PDF]

Sci Rep
Lavrikova AY, Aebi A, Avino F, Dubois EY, Schneider BL, Furno I.   +5 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

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

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

Insertion-Site Proximity to AAV Inverted Terminal Repeats Increases Plasmid Recombination. [PDF]

Int J Mol Sci
Makarenko M, Semicheva D, Fishman V.
europepmc   +1 more source

Modified Plasmids and Inverted Terminal Repeats Enhance Adeno-Associated Virus Production and Performance. [PDF]

Int J Mol Sci
Donohue N, Bogdanovic A, Conheady J, Davin S, Barron N, Glennon B.   +5 more
europepmc   +1 more source

Exploration of genes and identification of evolutionary evidence in adeno-associated viruses. [PDF]

J Microbiol
Lee C, Min J, Lim S, Park A, Kwak S, Hwang S, Park S, Jang YS, Cha SY, Cho SG, Lee JY.   +10 more
europepmc   +1 more source

Adeno-Associated Virus Gene Therapy: Is There a Risk of Insertional Mutagenesis? [PDF]

Toxicol Pathol
Batty P.
europepmc   +1 more source

Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A. [PDF]

Haemophilia
Raheja P, Rangarajan S, Lester W, Madan B, Pierce GF, Symington E, Millar C, Osmond D, Li M, Chavele KM.   +9 more
europepmc   +1 more source