Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping-Reference-Cited by-同舟云学术

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

Published:2021-09-01 Issue:17-18 Volume:32 Page:882-894
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Gushchina Liubov V.¹,Frair Emma C.¹,Rohan Natalie¹,Bradley Adrienne J.¹,Simmons Tabatha R.¹,Chavan Hemantkumar D.²,Chou Hsin-Jung³,Eggers Michelle³,Waldrop Megan A.¹⁴,Wein Nicolas¹⁴,Flanigan Kevin M.¹⁴⁵

Affiliation:

1. The Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital.

2. Charles River Laboratories, Wilmington, Massachusetts, USA.

3. Audentes Therapeutics, San Francisco, California, USA.

4. Departments of Pediatrics, The Ohio State University, Columbus, Ohio, USA.

5. Departments of Neurology, The Ohio State University, Columbus, Ohio, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2020.286

Reference47 articles.

1. Current and emerging therapies for Duchenne muscular dystrophy and spinal muscular atrophy

2. Double-Target Antisense U7 snRNAs Promote Efficient Skipping of an Aberrant Exon in Three Human -Thalassemic Mutations

3. U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping

4. The special Sm core structure of the U7 snRNP: far-reaching significance of a small nuclear ribonucleoprotein

5. Spinal Muscular Atrophy: SMN2 Pre-mRNA Splicing Corrected by a U7 snRNA Derivative Carrying a Splicing Enhancer Sequence

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