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US 2026/0306670 A1Published application
Base editing approaches to treat abca4-associated stargardt disease
AssigneeMassachusetts Eye and Ear Infirmary
InventorsCaitlin Collin, Qin Liu
Life of this patent
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Abstract
Described herein are base editors (BEs)/single guide RNAs (sgRNAs) and their use in methods of treating recessive retinal disease caused by mutations in ABCA4.
Claims23
A composition comprising a single guide RNA (sgRNA) comprising a Protospacer Sequence comprising one of SEQ ID NOs:6 to 37, and optionally a corresponding base editor (BE), optionally an adenine base editor (ABE) or cytosine base editor (CBE).
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A method of altering a cell, comprising contacting the cell with a recombinant viral particle comprising:
- a nucleotide sequence encoding an sgRNA comprising a Protospacer Sequence comprising one of SEQ ID NOs:6 to 37, and a corresponding base editor (BE), optionally an adenine base editor (ABE) or cytosine base editor (CBE), or
- wherein said viral particle is capable of delivery to a non-dividing cell, and wherein said contacting results in correction of a pathogenic mutation in an ABCA4 allele in the cell.
Dependencies are inferred from the claim text.