Methods and compositions for treatment of myotonia

Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Carbohydrate doai

Reexamination Certificate

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C435S325000, C536S023100

Reexamination Certificate

active

07964570

ABSTRACT:
The present invention provides methods and compositions for the treatment of diseases associated with aberrant microsatellite expansions. Methods of the present invention comprise the use of recombinant adeno-associated virus vectors containing a transgene encoding at least one muscleblind protein. The present invention also provides an animal model for a disease associated with aberrant microsatellite expansion.

REFERENCES:
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Miller, et al., Recruitment of Human Muscleblind Proteins to (CUG)nExpansions Associated with Myotonic Dystrophy, The Embo Journal, 2000, vol. 19, No. 17 pp. 4439-4448.
Hartigan-O'Connor, et al., “Developments in Gene Therapy for Muscular Dystrophy”, Microscopy Research and Technique, 2000, 48:223-238.
International Search Report (Form PCT/ISA/220 (Jan. 2004).

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