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US 2022/0090084 A1Published application
Antisense-induced exon exclusion in myostatin
OwnerFrederick Joseph Schnell, Richard Keith Bestwickinventors
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Abstract
The present disclosure relates to antisense oligomers and related compositions and methods for decreasing the expression of functional human myostatin and methods for treating muscular dystrophy and related disorders and more specifically relates to inducing exclusion of myostatin exon 2 and thereby reducing levels of myostatin protein.
Claims1
An antisense oligomer compound comprising a sequence selected from SEQ ID NOS:
- 2-5, 10-15, and 17-30, wherein X is independently selected from uracil (U) or thymine (T), comprising:
- at least one subunit that is a nucleotide analog having (i) a modified internucleoside linkage (ii) a modified sugar moiety, or (iii) a combination of the foregoing.
Dependencies are inferred from the claim text.