Compositions and methods comprising programmable snrnas for rna editing
AssigneeRegeneron Pharmaceuticals, Inc.
InventorsRea LARDELLI MARKMILLER, Ranjan BATRA, Rachel A. ADAMS, Alberto CARRENO
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Abstract
SnRNA systems comprising RNA binding sequences for RNA editing are disclosed herein.
Claims40
An RNA-targeting nucleic acid molecule comprising a small nuclear RNA (snRNA) molecule, wherein the snRNA molecule comprises at least one targeting sequence, wherein the targeting sequence has at least one base-pairing mismatch, an Sm binding domain (SmBD), and an snRNA stem loop.
An RNA-targeting nucleic acid molecule comprising an snRNA system (snRNA), wherein the snRNA system comprises at least one targeting sequence, and an Sm binding domain (SmBD), and an snRNA stem loop, wherein the binding sequence has extensive complementarity to a target RNA.
Dependencies are inferred from the claim text.