faculty

Jeffrey Chamberlain

jsc5@uw.edu

University of Washington, 

Cell Signaling & Cell/Environment Interactions

Developmental Biology, Stem Cells & Aging

Genetics, Genomics & Evolution

Gene therapy, muscular dystrophy, stem cells, CRISPR/Cas9

Faculty Contact Information

Building: K wing HSB Room: K243b Box: 357720 Phone: 206-221-5363 Alt Phone: 206-616-6645

Lab Information

Location: UW HSB Building: K wing Room: K233, K227 Box: 357720 Phone: 206-221-5412

Accepting Students For:

Rotation, Autumn
Rotation, Spring
Rotation, Winter
Permanent

Publications

Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice.

Kennedy TL, Guiraud S, Edwards B, Squire S, Moir L, Babbs A, Odom G, Golebiowski D, Schneider J, Chamberlain JS, Davies KE.

Molecular therapy. Methods & clinical development. 2018; 11:92-105.

PubMed [journal]
PMID:
30417024
PMCID:
PMC6216100

Non-invasive tracking of disease progression in young dystrophic muscles using multi-parametric MRI at 14T.

Park JS, Vohra R, Klussmann T, Bengtsson NE, Chamberlain JS, Lee D.

PloS one. 2018; 13(10):e0206323.

PubMed [journal]
PMID:
30365532
PMCID:
PMC6203357

Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy.

Ho PP, Lahey LJ, Mourkioti F, Kraft PE, Filareto A, Brandt M, Magnusson KEG, Finn EE, Chamberlain JS, Robinson WH, Blau HM, Steinman L.

Proceedings of the National Academy of Sciences of the United States of America. 2018; 115(39):E9182-E9191.

PubMed [journal]
PMID:
30181272
PMCID:
PMC6166850

Syntrophin binds directly to multiple spectrin-like repeats in dystrophin and mediates binding of nNOS to repeats 16-17.

Adams ME, Odom GL, Kim MJ, Chamberlain JS, Froehner SC.

Human molecular genetics. 2018; 27(17):2978-2985.

PubMed [journal]
PMID:
29790927
PMCID:
PMC6097012

Cas9 immunity creates challenges for CRISPR gene editing therapies.

Crudele JM, Chamberlain JS.

Nature communications. 2018; 9(1):3497.

PubMed [journal]
PMID:
30158648
PMCID:
PMC6115392

Research Summary

Our lab studies mechanisms leading to the muscular dystrophies, the function of dystrophin (DMD, among the most common inherited diseases) and FKRP (LGMD2I), and approaches to therapy. Recent work has focused on the use of AAV vectors, as we discovered that they have a remarkable ability to deliver genes bodywide via the vasculature. Significant amelioration of muscular dystrophy is achieved using systemic AAV delivery of replacement genes, or by delivery of CRISPR/Cas9 components to induce gene editing. The lab also studies myogenic stem cells, including iPSCs, and their use in therapy.