Honors
1995, Faculty Recognition Award,
University of Michigan
1995, Gift of Hope
Award, Jones Intercable, Inc.
1994, Leadership
Award, Service Merchandise Inc.
Research
Our
group is focused on understanding and developing treatments for
the
muscular dystrophies and other disorders of muscle.
These studies
center on the Duchenne muscular dystrophy (DMD) gene (dystrophin) and
explore the expression and function of this gene and its protein
isoforms. A major area of focus involves the development of viral
vectors to deliver dystrophin or other genes to muscle for gene
therapy. These vectors are being tested for safety and for their
ability to halt or reverse the dystrophic phenotype. The primary vector
being studied is adeno-associated virus (AAV), which efficiently
transfers genes to skeletal muscle and heart. Methods are being
developed for whole body systemic delivery, and a human clinical trial
is being planned. We are also studying adenoviral vectors, particularly
fully 'gutted' adenoviruses, and modified, replication-defective
vectors. An additional area of study is an investigation of myogenic
stem cells and their potential use in ex vivo gene therapy following
transduction of isolated cells with lentiviral vectors. Our efforts to
transfer genes to muscle are also being adapted for genetic and
non-genetic muscle wasting disorders. Dr. Chamberlain is currently
director of the Senator Paul D. Wellstone Muscular Dystrophy
Cooperative Research Center at the University of Washington School of
Medicine.
Selected
Publications
Abmayr S, Gregorevic P,
Allen JM, Chamberlain JS (2005) Phenotypic
improvement of dystrophic muscles by rAAV/microdystrophin vectors is
augmented
by Igf1 codelivery. Mol Ther 12, 441-450.
Balcaitis S, Weinstein
JR, Li S, Chamberlain JS, Moller T (2005)
Lentiviral transduction of microglial cells. Glia 50, 48-55.
Blankinship MJ,
Gregorevic P, Chamberlain JS (2005) Gene Therapy
Strategies for Duchenne Muscular Dystrophy Utilizing Recombinant
Adeno-associated Virus Vectors. Mol Ther.
Gregorevic P,
Chamberlain JS (2005) Functional enhancement of skeletal
muscle by gene transfer. Phys Med Rehabil Clin N Am 16, 875-887,
vii-viii.
Lai Y, Yue Y, Liu M,
Ghosh A, Engelhardt JF, Chamberlain JS, Duan D
(2005) Efficient in vivo gene expression by trans-splicing
adeno-associated
viral vectors. Nat Biotechnol 23, 1435-1439.
Abmayr S, Crawford RW,
Chamberlain JS (2004) Characterization of ARC,
apoptosis repressor interacting with CARD, in normal and
dystrophin-deficient
skeletal muscle. Hum Mol Genet 13, 213-221.
Bachrach E, Li S, Perez
AL, Schienda J, Liadaki K, Volinski J, Flint A,
Chamberlain J, Kunkel LM (2004) Systemic delivery of human
microdystrophin to
regenerating mouse dystrophic muscle by muscle progenitor cells. Proc
Natl Acad
Sci U S A 101, 3581-3586.
Blankinship MJ,
Gregorevic P, Allen JM, Harper SQ, Harper H, Halbert CL,
Miller DA, Chamberlain JS (2004) Efficient transduction of skeletal
muscle
using vectors based on adeno-associated virus serotype 6. Mol Ther 10,
671-678.
Chamberlain JS (2004)
Cachexia in cancer--zeroing in on myosin. N Engl J
Med 351, 2124-2125.
Friedrich O, Both M,
Gillis JM, Chamberlain JS, Fink RH (2004)
Mini-dystrophin restores L-type calcium currents in skeletal muscle of
transgenic mdx mice. J Physiol 555, 251-265.
Ge Y, Molloy MP,
Chamberlain JS, Andrews PC (2004) Differential
expression of the skeletal muscle proteome in mdx mice at different
ages.
Electrophoresis 25, 2576-2585.
Gregorevic P,
Blankinship MJ, Allen JM, Crawford RW, Meuse L, Miller DG,
Russell DW, Chamberlain JS (2004) Systemic delivery of genes to
striated
muscles using adeno-associated viral vectors. Nat Med 10, 828-834.
Gregorevic P,
Blankinship MJ, Chamberlain JS (2004) Viral vectors for
gene transfer to striated muscle. Curr Opin Mol Ther 6, 491-498.
Chamberlain JS, Barjot
C, Scott J (2003) Packaging cell lines for
generating replication-defective and gutted adenoviral vectors. Methods
Mol Med
76, 153-166.
Ge Y, Molloy MP,
Chamberlain JS, Andrews PC (2003) Proteomic analysis of
mdx skeletal muscle: Great reduction of adenylate kinase 1 expression
and
enzymatic activity. Proteomics 3, 1895-1903.
Gregorevic P,
Chamberlain JS (2003) Gene therapy for muscular dystrophy
- a review of promising progress. Expert Opin Biol Ther 3, 803-814.
Box 357350 