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US 12,180,499 B2Granted
Compositions and methods for impeding transcription of expanded microsatellite repeats
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsEric Tzy-Shi Wang, Tanvi Saxena, Belinda Pinto, John Andrew Berglund
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Abstract
Disclosed herein are compositions and methods for treating diseases characterized by expanded microsatellite repeats by impeding or inhibiting transcription of expanded microsatellite repeats.
Claims2
An adeno-associated virus (AAV) vector, an AAV virion or an AAV viral particle comprising:
- a viral capsid;
- a nucleic acid encoding a deactivated S. aureus Cas9 (dSaCas9) that is not fused to a transcriptional repressor;
- and
- a nucleic acid encoding a CTG repeat-targeting guide RNA (gRNA).
1 more element
Dependencies are inferred from the claim text.