Invention Application
- Patent Title: METHODS AND SYSTEMS FOR CORRECTING MUTATIONS IN PRPH2
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Application No.: US18946509Application Date: 2024-11-13
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Publication No.: US20250090690A1Publication Date: 2025-03-20
- Inventor: Yi-Ting Tsai , Salvatore M. Caruso , Bruna Lopes da Costa , Stephen H. Tsang , Peter M.J. Quinn
- Applicant: The Trustees of Columbia University in the City of New York
- Applicant Address: US NY New York
- Assignee: The Trustees of Columbia University in the City of New York
- Current Assignee: The Trustees of Columbia University in the City of New York
- Current Assignee Address: US NY New York
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61K38/17 ; A61P27/02 ; C12N9/12 ; C12N9/22 ; C12N15/11
Abstract:
The present disclosure provides systems, methods, and compositions for prime-editing modification of c.828 splice site mutations in the peripherin-2 gene. Particularly the present disclosure provides systems, methods, and compositions for correcting one or more disease-causing splice site mutations selected from: c.828+3A>T, c.828+1G>A, c.828+2T>C, c.828+1G>T, and combinations thereof.
Information query
