Noura S. Abul-Husn,Omri Gottesman,Alexander Li,Xiping Cheng,Yurong Xin,Evangelos Pefanis,Suzanne Hartford,Jesper Gromada,Frederick E. Dewey,Aris Baras,Alan Shuldiner
申请号:
US15875514
公开号:
US20180216104A1
申请日:
2018.01.19
申请国别(地区):
US
年份:
2018
代理人:
摘要:
Provided are compositions related to HSD17B13 variants, including isolated nucleic acids and proteins related to variants of HSD17B13, and cells comprising those nucleic acids and proteins. Also provided are methods related to HSD17B13 variants. Such methods include methods for modifying a cell through use of any combination of nuclease agents, exogenous donor sequences, transcriptional activators, transcriptional repressors, and expression vectors for expressing a recombinant HSD17B13 gene or a nucleic acid encoding an HSD17B13 protein. Also provided are therapeutic and prophylactic methods for treating a subject having or at risk of developing chronic liver disease.