Invention Application
- Patent Title: NUCLEOTIDES COMPRISING CASPHI MODIFIED FOR NUCLEAR TARGETING
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Application No.: PCT/US2022/041561Application Date: 2022-08-25
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Publication No.: WO2023028254A2Publication Date: 2023-03-02
- Inventor: REX, Tonia S. , BACKSTROM, Jon R.
- Applicant: VANDERBILT UNIVERSITY
- Applicant Address: 305 Kirkland Hall
- Assignee: VANDERBILT UNIVERSITY
- Current Assignee: VANDERBILT UNIVERSITY
- Current Assignee Address: 305 Kirkland Hall
- Agency: DECKER, Mandy Wilson et al.
- Priority: US63/237,077 2021-08-25
- Main IPC: C12N15/09
- IPC: C12N15/09 ; C12N15/11 ; C12N15/63 ; C12N15/79 ; C12N15/85 ; C12N15/86 ; C12N15/87 ; C12N15/90 ; A61K48/00 ; C12N15/00 ; C12N15/102 ; C12N15/635 ; C12N2310/20 ; C12N2750/14143
Abstract:
The presently-disclosed subject matter relates to a compositions and methods that makes use of a CasΦ(CasPhi) that has been modified for effective nuclear targeting. In particular, certain embodiments of the presently-disclosed subject matter relate to unique nucleic acid molecules, compositions, and methods for delivery of CasPhi to the nucleus to effectively modulate expression or otherwise target of a gene of interest. A nucleic acid disclosed herein includes a nucleotide encoding a CasPhi, wherein there are no amino acid substitutions, or wherein proline residues at amino acids 749 and 753 have been substituted; a nucleotide encoding an amino-terminal linker connected to the amino-terminal end of the CasPhi; a nucleotide encoding a carboxy -terminal linker connected to the carboxy-terminal end of the CasPhi; and a nucleotide encoding a nuclear localization signal (NLS) downstream from the CasPhi and carboxy-terminal linker.
Information query
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