US2016160217A1PendingUtilityA1
Compositions and methods for characterizing and treating muscular dystrophy
Est. expiryApr 11, 2032(~5.7 yrs left)· nominal 20-yr term from priority
C12N 15/1137C12Q 2600/106C12N 2310/11C12N 15/1138C12N 15/111C12N 2310/346C12N 2310/3233C12N 2310/315C12Q 2600/158C12N 5/0658C12N 2320/12C12N 15/113C12N 2320/30A61P 21/00C12N 2310/341C12Q 1/6883A01K 67/0271C12N 2310/322
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Claims
Abstract
Compositions and methods for identifying new treatments for Facioscapulohumeral muscular dystrophy (FSHD), and uses thereof.
Claims
exact text as granted — not AI-modified1 .- 21 . (canceled)
22 . A method of treating Facioscapulohumeral muscular dystrophy (FSHD) in a subject, the method comprising administering to the subject one or more inhibitory nucleic acids targeting one or more of SLC34A2, TRIM49, TRIM43, CD177, NAAA, HSPA6, TC2N, or CD34.
23 . A method of treating FSHD in a subject, the method comprising administering to the subject two or more inhibitory nucleic acids targeting two or more of SLC34A2, TRIM49, TRIM43, PRAMEF1, CD177, NAAA, HSPA6, TC2N, or CD34.
24 . The method of claim 22 or 23 , wherein the inhibitory nucleic acid is a double-stranded RNA, siRNA, shRNA, or antisense oligonucleotide.
25 . The method of claim 24 , wherein the antisense oligonucleotide is a morpholino oligonucleotide.Join the waitlist — get patent alerts
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