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US 2026/0061074 A1Published application
Genetic constructs for gene editing
AssigneeSarepta Therapeutics, Inc.
InventorsAmi M. KABADI, Trenton FRISBIE, Karen BULAKLAK, Chang LIU
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Abstract
The present disclosure provides methods and compositions concerning the disclosed genetic constructs, including those that target mutated portions of a dystrophin gene for excision, thereby restoring functional dystrophin protein expression. The disclosure further provides methods and compositions for treating Duchenne muscular dystrophy.
Claims20
-67. (canceled)
A genetic construct, comprising:
- i) a first inverted terminal repeat (ITR) nucleotide sequence;
- ii) an RNA polymerase II (Pol II)-driven promoter operably linked to a transgene;
- iii) a first RNA polymerase III (Pol III)-driven promoter operably linked to a first guide RNA (gRNA) nucleotide sequence as set forth in SEQ ID NO:
- 14;
- iv) a second Poll III-driven promoter operably linked to a second gRNA nucleotide sequence as set forth in SEQ ID NO:
- 31,
- wherein the first and second Pol III-driven promoters and the first and second gRNAs are in a reverse orientation to the Pol II-driven promoter and the transgene;
- and
- v) a second ITR nucleotide sequence,
- wherein said genetic construct is encoded in a single polynucleotide.
7 more elements
Dependencies are inferred from the claim text.