Disrupting the Molecular Pathway in Myotonic Dystrophy
2021 ◽
Vol 22
(24)
◽
pp. 13225
Keyword(s):
Myotonic dystrophy is the most common muscular dystrophy in adults. It consists of two forms: type 1 (DM1) and type 2 (DM2). DM1 is associated with a trinucleotide repeat expansion mutation, which is transcribed but not translated into protein. The mutant RNA remains in the nucleus, which leads to a series of downstream abnormalities. DM1 is widely considered to be an RNA-based disorder. Thus, we consider three areas of the RNA pathway that may offer targeting opportunities to disrupt the production, stability, and degradation of the mutant RNA.
Keyword(s):
Keyword(s):
2005 ◽
Vol 43
(5)
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pp. e23-e23
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Keyword(s):
1997 ◽
Vol 20
(2)
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pp. 232-234
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Keyword(s):
1997 ◽
Vol 17
(4)
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pp. 2090-2098
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2019 ◽
Vol 60
(10)
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pp. 3636
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