To visit this center's site, go to http://depts.washington.edu/mdcrc/
Center Abstract
Project 1: Pre-Clinical Studies and Development of a Phase 1 Clinical Trial for Gene Transfer in DMD
PIs: Jeffrey S. Chamberlain, Ph.D. ; Stephen Tapscott, M.D., Ph.D. Project Abstract
Project 2: Testing AAV Vectors in a Canine DMD Model
PI: Christian Kuhr, M.D. Project Abstract
Project 3: Optimizing Muscle-Specific Regulatory Gene Cassettes for Human Muscular Gene Therapy
PI: Stephen D. Hauschka, Ph.D . Project Abstract
Project 4: Molecular Pathogenesis of Myotonic Dystrophy
PI: Stephen Tapscott, M.D., Ph.D. Project Abstract
Core A: Administrative Core
PI: Jeffrey S. Chamberlain, Ph.D. Core Abstract
Core B: Viral Vector Laboratory
Publications
Cho DH, Tapscott SJ. Myotonic dystrophy: Emerging mechanisms for DM1 and DM2. Biochim Biophys Acta, 2006. (Epub ahead of print) Gaedigk R, Law DJ, Fitzgerald-Gustafson KM, McNulty SG, Nsumu NN, Modrcin AC, Rinaldi RJ, Pinson D, Fowler SC, Bilgen M, Burns J, Hauschka SD, White RA. Improvement in survival and muscle function in an mdx/utrn(-/-) double mutant mouse using a human retinal dystrophin transgene. Neuromuscul Disord 16(3):192-203, 2006. Gregorevic P, Allen JM, Minami E, Blankinship MJ, Haraguchi M, Meuse L, Finn E, Adams ME, Froehner SC, Murry CE, and JS Chamberlain. rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 12(7): 787-9, 2006. Blankinship MJ, Gregorevic P, and JS Chamberlain. Gene therapy strategies for duchenne muscular dystrophy utilizing recombinant adeno-associated virus vectors. Mol Ther 13: 241-9, 2006. Han JJ, Carter GT, Ra JJ, Abresch RT, Chamberlain JS, and LR Robinson. Electromyographic studies in mdx and wild-type C57 mice. Muscle Nerve 33: 208-14, 2006. Cho DH, Thienes CP, Mahoney SE, Analau E, Filippova GN, Tapscott SJ. Antisense transcription and heterochromatin at the DM1 CTG repeats are constrained by CTCF. Mol Cell 20(3):483-9, 2005. Li S, Kimura E, Fall BM, Reyes M, Angello JC, Welikson R, Hauschka SD, and JS Chamberlain. Stable transduction of myogenic cells with lentiviral vectors expressing a minidystrophin. Gene Ther 12: 1099-108, 2005. Liu M, Yue Y, Harper SQ, Grange RW, Chamberlain JS, and D Duan. Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Mol Ther 11: 245-56, 2005. Blankinship MJ, Gregorevic P, Allen JM, Harper SQ, Harper H, Halbert CL, Miller DA, and JS Chamberlain. Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol Ther 10: 671-8, 2004. Dell'Agnola C, Wang Z, Storb R, Tapscott SJ, Kuhr CS, Hauschka SD, Lee RS, Sale GE, Zellmer E, Gisburne S, Bogan J, Kornegay JN, Cooper BJ, Gooley TA, and MT Little. Hematopoietic stem cell transplantation does not restore dystrophin expression in Duchenne muscular dystrophy dogs. Blood 104: 4311-8, 2004. Gregorevic P, Blankinship MJ, Allen JM, Crawford RW, Meuse L, Miller DG, Russell DW, and JS Chamberlain. Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med 10: 828-34, 2004. Gregorevic P, Blankinship MJ, and JS Chamberlain. Viral vectors for gene transfer to striated muscle. Curr Opin Mol Ther 6: 491-8, 2004. Baron F, Storb R, and M Little. Hematopoietic Cell Transplantation: Five Decades of Progress. Arch Med Res 34: 528-44, 2003.
Cho DH, Tapscott SJ. Myotonic dystrophy: Emerging mechanisms for DM1 and DM2. Biochim Biophys Acta, 2006. (Epub ahead of print)
Gaedigk R, Law DJ, Fitzgerald-Gustafson KM, McNulty SG, Nsumu NN, Modrcin AC, Rinaldi RJ, Pinson D, Fowler SC, Bilgen M, Burns J, Hauschka SD, White RA. Improvement in survival and muscle function in an mdx/utrn(-/-) double mutant mouse using a human retinal dystrophin transgene. Neuromuscul Disord 16(3):192-203, 2006.
Gregorevic P, Allen JM, Minami E, Blankinship MJ, Haraguchi M, Meuse L, Finn E, Adams ME, Froehner SC, Murry CE, and JS Chamberlain. rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 12(7): 787-9, 2006.
Blankinship MJ, Gregorevic P, and JS Chamberlain. Gene therapy strategies for duchenne muscular dystrophy utilizing recombinant adeno-associated virus vectors. Mol Ther 13: 241-9, 2006.
Han JJ, Carter GT, Ra JJ, Abresch RT, Chamberlain JS, and LR Robinson. Electromyographic studies in mdx and wild-type C57 mice. Muscle Nerve 33: 208-14, 2006.
Cho DH, Thienes CP, Mahoney SE, Analau E, Filippova GN, Tapscott SJ. Antisense transcription and heterochromatin at the DM1 CTG repeats are constrained by CTCF. Mol Cell 20(3):483-9, 2005.
Li S, Kimura E, Fall BM, Reyes M, Angello JC, Welikson R, Hauschka SD, and JS Chamberlain. Stable transduction of myogenic cells with lentiviral vectors expressing a minidystrophin. Gene Ther 12: 1099-108, 2005.
Liu M, Yue Y, Harper SQ, Grange RW, Chamberlain JS, and D Duan. Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Mol Ther 11: 245-56, 2005.
Blankinship MJ, Gregorevic P, Allen JM, Harper SQ, Harper H, Halbert CL, Miller DA, and JS Chamberlain. Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol Ther 10: 671-8, 2004.
Dell'Agnola C, Wang Z, Storb R, Tapscott SJ, Kuhr CS, Hauschka SD, Lee RS, Sale GE, Zellmer E, Gisburne S, Bogan J, Kornegay JN, Cooper BJ, Gooley TA, and MT Little. Hematopoietic stem cell transplantation does not restore dystrophin expression in Duchenne muscular dystrophy dogs. Blood 104: 4311-8, 2004.
Gregorevic P, Blankinship MJ, Allen JM, Crawford RW, Meuse L, Miller DG, Russell DW, and JS Chamberlain. Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med 10: 828-34, 2004.
Gregorevic P, Blankinship MJ, and JS Chamberlain. Viral vectors for gene transfer to striated muscle. Curr Opin Mol Ther 6: 491-8, 2004.
Baron F, Storb R, and M Little. Hematopoietic Cell Transplantation: Five Decades of Progress. Arch Med Res 34: 528-44, 2003.