Journal
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE
Volume 1772, Issue 2, Pages 243-262Publisher
ELSEVIER
DOI: 10.1016/j.bbadis.2006.09.007
Keywords
dystrophin; utrophin; adeno-associated virus; exon skipping; antisensc oligonucleotide
Funding
- NHLBI NIH HHS [T32 HL07828, T32 HL007828] Funding Source: Medline
- NIAMS NIH HHS [R01 AR044533, R37 AR040864, AR44533] Funding Source: Medline
- NICHD NIH HHS [U54 HD047175] Funding Source: Medline
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Much progress has been made over the past decade elucidating the molecular basis for a variety of muscular dystrophies (MDs). Accordingly, there are examples of mouse models of MD whose disease progression has been halted in large part with the use of viral vector technology. Even so, we must acknowledge significant limitations of present vector systems that must be overcome prior to successful treatment of humans with such approaches. This review will present a variety of viral-mediated therapeutic strategies aimed at counteracting the muscle-wasting symptoms associated with muscular dystrophy. We include viral vector systems used for muscle gene transfer, with a particular emphasis on adeno-associated virus. Findings of several encouraging studies focusing on repair of the mutant dystrophin gene are also included. Lastly, we present a discussion of muscle compensatory therapeutics being considered that include pathways involved in the up-regulation of utrophin, promotion of cellular adhesion, enhancement of muscle mass, and antagonism of the inflammatory response. Considering the complexity of the muscular dystrophies, it appears likely that a multilayered approach tailored to a patient sub-group may be warranted in order to effectively contest the progression of this devastating disease. (c) 2006 Elsevier B.V. All rights reserved.
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