SAREPTA THERAPEUTICS INC.;THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES;UNIVERSITY OF MARYLAND;THE PROGERIA RESEARCH FOUNDATION
发明人:
ERDOS, Michael R.,CAO, Kan,KOLE, Ryszard,BESTWICK, Richard Keith,COLLINS, Francis S.,GORDON, Leslie B.
申请号:
CO2018012873
公开号:
CO2018012873A2
申请日:
2018.11.27
申请国别(地区):
CO
年份:
2020
代理人:
摘要:
Anti cleavage oligonucleotides were provided to LMNA to reduce the expression of one or more isomerized acrylonitrile butadiene butadiene styrene copolymers bound in an abnormal manner.Se proporcionan oligonucleótidos antisentido dirigidos a LMNA para la reducción de la expresión de una o más isoformas de mARN LMNA empalmadas de forma aberrante que codifican progerina.