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:
FRANCIS S. COLLINS,KAN CAO,LESLIE B. GORDON,MICHAEL R. ERDOS,RICHARD KEITH BESTWICK,RYSZARD KOLE
申请号：
BR112018072279
公开号：
BR112018072279A2
申请日:
2017.04.28
申请国别(地区):
BR
年份:
2019
代理人:
摘要：
antisense oligonucleotides targeting 1mna are provided to reduce expression of one or more aberrantly split progenin encoding 1mna mrna isoforms.são fornecidos oligonucleotídeos antissentido tendo como alvo lmna para reduzir expressão de uma ou mais isoformas de mrna de lmna divididas aberrantemene que codificam progerina.
