Výsledky vyhledávání - "Natalie Rohan"

Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping

Autor: Michelle Eggers, Nicolas Wein, Hemantkumar D Chavan, Kevin M. Flanigan, Adrienne J Bradley, Liubov V Gushchina, Emma C Frair, Hsin-Jung Chou, Megan A. Waldrop, Tabatha R. Simmons, Natalie Rohan

Publikováno v: Human Gene Therapy. 32:882-894

Therapeutic exon skipping as a treatment for Duchenne muscular dystrophy (DMD) has largely concentrated on the delivery of antisense oligomers to treat out-of-frame exon deletions. Here we report o...

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::5e6a19be41010c71837d9b049f88e3ad
https://doi.org/10.1089/hum.2020.286

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Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce

Autor: Liubov V, Gushchina, Emma C, Frair, Natalie, Rohan, Adrienne J, Bradley, Tabatha R, Simmons, Hemantkumar D, Chavan, Hsin-Jung, Chou, Michelle, Eggers, Megan A, Waldrop, Nicolas, Wein, Kevin M, Flanigan

Publikováno v: Human gene therapy. 32(17-18)

Therapeutic exon skipping as a treatment for Duchenne muscular dystrophy (DMD) has largely concentrated on the delivery of antisense oligomers to treat out-of-frame exon deletions. Here we report on the preclinical development of an adeno-associated

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::95fe550ec7788f1301027bef02b71ee5
https://pubmed.ncbi.nlm.nih.gov/33406986

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