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US 12,629,428 B2Granted
AAV vector-mediated deletion of large mutational hotspot for treatment of Duchenne muscular dystrophy
AssigneeDuke University
InventorsCharles A. Gersbach, Karen Bulaklak, Jacqueline N. Robinson-Hamm
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Abstract
Disclosed herein are therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use.
Claims17
A DNA targeting system comprising a single vector encoding:
- (a) a first guide RNA (gRNA) molecule targeting intron 44 of dystrophin;
- (b) a second gRNA molecule targeting intron 55 of dystrophin;
- and
- (c) a Clustered Regularly Interspaced Short Palindromic Repeats Associated 9 (Cas9) protein;
- and
- (d) one or more Cas9 gRNA scaffolds,
- wherein the single vector comprises:
- a first promoter comprising a U6 promoter and driving expression of the first gRNA molecule;
- a second promoter driving expression of the Cas9 protein;
- and
- a third promoter comprising a H1 promoter driving expression of the second gRNA molecule,
- wherein the first promoter is upstream of the second promoter and drives expression in a direction opposite to the second and third promoters.
9 more elements
Dependencies are inferred from the claim text.