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US 2025/0084420 A1Published application
Compositions and methods for treating cmt1a or cmt1e diseases with rnai molecules targeting pmp22
AssigneesUniversite De Montpellier, Institut National De La Sante Et De La Recherche Medicale (Inserm)
InventorNicolas TRICAUD
Life of this patent
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Abstract
RNA interferent (RNAi) molecules that inhibit a PMP22 protein expression and/or activity by targeting exon 5 of a nucleic acid sequence encoding the PMP22 protein, and the prevention and treatment of the Charcot-Marie-Tooth type 1A or 1E diseases in which an adeno-associated virus (AAV) vector including an RNAi molecule is administered.
Claims12
1-11. (canceled)
An adeno-associated virus (AAV) vector comprising an RNAi molecule comprising an antisense oligonucleotide of sequence SEQ ID NO:
- 61, and/or an oligonucleotide coding for an antisense oligonucleotide of sequence SEQ ID NO:
- 61.
Dependencies are inferred from the claim text.