MATERIALS AND METHODS FOR TREATMENT OF SPINOCEREBELLAR ATAXIA TYPE 2 (SCA2) AND OTHER SPINOCEREBELLAR ATAXIA TYPE 2 PROTEIN (ATXN2) GENE RELATED CONDITIONS OR DISORDERS
Ante Sven LUNDBERG,Samarth KULKARNI,Lawrence KLEIN,Hari Kumar PADMANABHAN
申请号：
US16487409
公开号：
US20200216857A1
申请日:
2018.02.21
申请国别(地区):
US
年份:
2020
代理人:
摘要：
The present disclosure provides materials and methods for treating a patient with one or more conditions or disorders associated with ATXN2 whether ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Spinocerebellar ataxia type 1 (SCA2). Also provided are materials and methods for editing a ATXN2 gene in a cell by genome editing. The present disclosure also provides materials and methods for altering the contiguous genomic sequence of a ATXN2 gene in a cell. In addition, the present disclosure provides one or more gRNAs for editing a ATXN2 gene. Also provided are therapeutics comprising at least one or more gRNAs for editing a ATXN2 gene. In addition, the present disclosure provides therapeutics for treating patients with a ATXN2 related condition or disorder.
