Compositions and methods for treatment of angelman syndrome
AssigneeThe Regents of the University of California
InventorsDavid J. SEGAL, Ulrika BEITNERE, Henriette O'GEEN
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Abstract
Compositions of a Cas13 protein and a guide RNA that targets a portion of a Ube3a-ATS transcript are provided herein, along with vectors that can continuously produce such compositions in cells, and cells that contain such vectors. These compositions, vectors, and cells can be used in methods for cleaving a portion of a Ube3a-ATS antisense RNA transcript and unsilencing the paternal UBE3A gene, as well as methods for treating Angelman Syndrome without impairing expression of Snord116 and/or Snord115.
Claims58
A composition comprising a Cas13 protein and a guide RNA that targets a Ube3a-ATS transcript.
A vector comprising a polynucleotide sequence encoding a Cas13 protein and a polynucleotide sequence encoding a guide RNA that targets a Ube3a-ATS transcript.
Dependencies are inferred from the claim text.