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Therapeutics development for triplet repeat expansion diseases

✍ Scribed by Di Prospero, Nicholas A.; Fischbeck, Kenneth H.

Book ID
109952186
Publisher
Nature Publishing Group
Year
2005
Tongue
English
Weight
391 KB
Volume
6
Category
Article
ISSN
1471-0056

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Expansions of unstable trinucleotide repeats cause at least 15 inherited neurologic diseases. Here we review what has been learned of three neuromuscular diseases caused by this type of mutation. X-linked spinal and bulbar muscular atrophy is a motor neuronopathy caused by a CAG repeat expansion in