J
Joel R. Chamberlain
Researcher at University of Washington
Publications - 26
Citations - 2055
Joel R. Chamberlain is an academic researcher from University of Washington. The author has contributed to research in topics: RNase P & RNase PH. The author has an hindex of 18, co-authored 26 publications receiving 1885 citations. Previous affiliations of Joel R. Chamberlain include University of Michigan.
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Journal ArticleDOI
Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.
Niclas E. Bengtsson,John K. Hall,Guy L. Odom,Michael Phelps,Colin Andrus,R. David Hawkins,Stephen D. Hauschka,Joel R. Chamberlain,Jeffrey S. Chamberlain +8 more
TL;DR: It is demonstrated that AAV-mediated muscle-specific gene editing has significant potential for therapy of neuromuscular disorders and systemic administration of the vectors results in widespread expression of dystrophin in both skeletal and cardiac muscles.
Journal ArticleDOI
Gene targeting in stem cells from individuals with osteogenesis imperfecta.
Joel R. Chamberlain,Ulrike Schwarze,Pei Rong Wang,Roli K. Hirata,Kurt D. Hankenson,James M. Pace,Robert A. Underwood,Kit M. Song,Michael D. Sussman,Peter H. Byers,David W. Russell +10 more
TL;DR: Adeno-associated virus vectors are used to disrupt dominant-negative mutant COL1A1 collagen genes in MSCs from individuals with the brittle bone disorder osteogenesis imperfecta, demonstrating successful gene targeting in adult human stem cells.
Journal ArticleDOI
Purification and characterization of the nuclear RNase P holoenzyme complex reveals extensive subunit overlap with RNase MRP
TL;DR: The degree of structural similarity between nuclear RNase P and RNase MRP suggests that some aspects of their functions in pre-tRNA and pre-rRNA processing pathways might overlap or be coordinated.
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Progress toward Gene Therapy for Duchenne Muscular Dystrophy.
TL;DR: Progress in the development of DMD gene therapy has been well documented in Molecular Therapy over the past 20 years and will be reviewed here to highlight prospects for success in the imminent human clinical trials planned by several groups.
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Targeted transgene insertion into human chromosomes by adeno-associated virus vectors.
TL;DR: AAV vectors are used to introduce large (>1 kb) functional transgene cassettes into the hypoxanthine phosphoribosyl transferase (HPRT) and Type I collagen loci in normal human fibroblasts and should prove useful both for functional genomic analysis in diploid human cells and for therapeutic gene targeting.