SCREENING OF CAS NUCLEASES FOR ALTERED NUCLEASE ACTIVITY

    公开(公告)号:US20250027134A1

    公开(公告)日:2025-01-23

    申请号:US18710260

    申请日:2022-11-15

    Abstract: Described herein is a method of screening for a nuclease with an altered nuclease activity comprising; a) forming a first compartmentalizing reaction comprising a carrier and a variant nuclease template nucleic acid comprising a coding region for a variant nuclease, and amplifying said coding region for said variant nuclease, to obtain variant nuclease encoding nucleic acid; b) forming a second compartmentalizing reaction comprising said variant nuclease encoding nucleic acid, and performing an in vitro transcription and translation reaction to obtain variant nuclease polypeptides; and c) forming a third compartmentalizing reaction comprising said variant nuclease polypeptides, and assaying said variant nuclease polypeptides for the altered nuclease activity of said nuclease.

    Compositions and Methods for Cell-Specific Expression of Target Genes

    公开(公告)号:US20250025575A1

    公开(公告)日:2025-01-23

    申请号:US18712552

    申请日:2022-11-23

    Abstract: Provided are polynucleotides comprising a transcriptional control element comprising a promoter sequence having at least 80% sequence identity to SEQ ID NO: 1. SEQ ID NO:2. SEQ ID NO:3 or SEQ ID NO:4 and a cell- or tissue-specific enhancer element. Also provided are compositions (e.g., vectors, viruses, host cells, and pharmaceutical compositions) comprising said polynucleotides, methods that are useful for expressing a target gene in a host cell, and methods that are useful for expressing a target gene in a subject in need thereof.

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