scholarly journals Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus

2015 ◽  
Vol 24 (20) ◽  
pp. 5880-5890 ◽  
Author(s):  
Yongping Yue ◽  
Xiufang Pan ◽  
Chady H. Hakim ◽  
Kasun Kodippili ◽  
Keqing Zhang ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Young Adult ◽  
Muscular Dystrophy ◽  
Adeno Associated Virus

Immunity to Adeno-Associated Virus-Mediated Gene Transfer in a Random-Bred Canine Model of Duchenne Muscular Dystrophy

2006 ◽  
Vol 0 (0) ◽  
pp. 061218064941001
Author(s):  
Zejing Wang ◽  
James M. Allen ◽  
Stanley R. Riddell ◽  
Paul Gregorevic ◽  
Rainer Storb ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transfer ◽  
Canine Model ◽  
Adeno Associated Virus

scholarly journals Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system

2020 ◽  
Vol 6 (8) ◽  
pp. eaay6812 ◽  
Author(s):  
Yu Zhang ◽  
Hui Li ◽  
Yi-Li Min ◽  
Efrain Sanchez-Ortiz ◽  
Jian Huang ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Genome Editing ◽  
Dystrophin Gene ◽  
Adeno Associated Virus ◽  
Guide Rnas ◽  
Dystrophin Expression ◽  
Cas9 Nuclease ◽  
High Doses

Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by mutations in the dystrophin gene (DMD). Previously, we applied CRISPR-Cas9–mediated “single-cut” genome editing to correct diverse genetic mutations in animal models of DMD. However, high doses of adeno-associated virus (AAV) are required for efficient in vivo genome editing, posing challenges for clinical application. In this study, we packaged Cas9 nuclease in single-stranded AAV (ssAAV) and CRISPR single guide RNAs in self-complementary AAV (scAAV) and delivered this dual AAV system into a mouse model of DMD. The dose of scAAV required for efficient genome editing were at least 20-fold lower than with ssAAV. Mice receiving systemic treatment showed restoration of dystrophin expression and improved muscle contractility. These findings show that the efficiency of CRISPR-Cas9–mediated genome editing can be substantially improved by using the scAAV system. This represents an important advancement toward therapeutic translation of genome editing for DMD.

Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD)

2015 ◽  
Vol 15 (4) ◽  
pp. 395-415 ◽  
Author(s):  
Klaudia Kawecka ◽  
Michael Theodoulides ◽  
Yalin Hasoglu ◽  
Susan Jarmin ◽  
Hanna Kymalainen ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transfer ◽  
Exon Skipping ◽  
Dystrophin Gene ◽  
Adeno Associated Virus

scholarly journals 499. Intravenous Delivery of a Novel Micro-Dystrophin Vector Prevented Muscle Deterioration in Young Adult Canine Duchenne Muscular Dystrophy Dogs

2016 ◽  
Vol 24 ◽  
pp. S198-S199 ◽  
Author(s):  
Chady Hakim ◽  
Xiufang Pan ◽  
Kasun Kodippili ◽  
Thais Blessa ◽  
Hsiao T. Yang ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Young Adult ◽  
Muscular Dystrophy
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