Ante Sven LUNDBERG,Samarth KULKARNI,Lawrence KLEIN,Hari Kumar PADMANABHAN
申请号：
US16487659
公开号：
US20200040061A1
申请日:
2018.02.16
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 SNCA whether ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Early Onset Parkinson's Disease (PARK1). Also provided are materials and methods for editing a SNCA gene in a cell by genome editing. The present disclosure also provides materials and methods for altering the contiguous genomic sequence of a SNCA gene in a cell. In addition, the present disclosure provides one or more gRNAs for editing a SNCA gene. Also provided are therapeutics comprising at least one or more gRNAs for editing a SNCA gene. In addition, the present disclosure provides therapeutics for treating patients with a SNCA related condition or disorder.
