MATERIALS AND METHODS FOR TREATMENT OF DYSTROPHIC EPIDERMOLYSIS BULLOSA (DEB) AND OTHER COLLAGEN TYPE VII ALPHA 1 CHAIN (COL7A1) GENE RELATED CONDITIONS OR DISORDERS
Ante Sven LUNDBERG,Samarth KULKARNI,Lawrence KLEIN,Hari Kumar PADMANABHAN
申请号:
US16487413
公开号:
US20190365929A1
申请日:
2018.02.14
申请国别(地区):
US
年份:
2019
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 COL7A1 whether ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Dystrophic Epidermolysis Bullosa (DEB). Also provided are materials and methods for editing a COL7A1 gene in a cell by genome editing. The present disclosure also provides materials and methods for altering the contiguous genomic sequence of a COL7A1 gene in a cell. In addition, the present disclosure provides one or more gRNAs for editing a COL7A1 gene. Also provided are therapeutics comprising at least one or more gRNAs for editing a COL7A1 gene. In addition, the present disclosure provides therapeutics for treating patients with a COL7A1 related condition or disorder.