Benjamin Stone AMIDON,James E. BROWNELL,James M. GAVIN,Erik M. KOENIG,Michael D. SINTCHAK,Peter G. SMITH
申请号：
US15170328
公开号：
US20170107579A1
申请日:
2016.06.01
申请国别(地区):
US
年份:
2017
代理人:
摘要：
The invention provides isolated nucleic acids molecules, designated UBA3, UAE, or UBA6, or other E1 enzyme variant nucleic acid molecules, which encode novel E1 enzyme variant proteins. The invention also provides antisense nucleic acid molecules, recombinant expression vectors containing UBA3, UAE, or UBA6, or other E1 enzyme variant nucleic acid molecules, host cells into which the expression vectors have been introduced, and nonhuman transgenic animals in which a UBA3, UAE, or UBA6, or other E1 enzyme variant gene has been introduced or disrupted. The invention still further provides isolated UBA3, UAE, or UBA6, or other E1 enzyme variant proteins, fusion proteins, antigenic peptides and anti-UBA3, UAE, or UBA6, or other E1 enzyme variant antibodies. The invention provides methods to identify agents that inhibit UBA3, UAE, or UBA6, or other E1 enzyme variant expression or activity. Diagnostic and therapeutic methods utilizing compositions of the invention are also provided.
