Inventors:
- Cambridge MA, US
Martin A. MAIER - Cambridge MA, US
Juan C. SALINAS - Cambridge MA, US
Shigeo MATSUDA - Cambridge MA, US
Scott P. LENTINI - Cambridge MA, US
Guo HE - Cambridge MA, US
Michelle H. JUNG - Cambridge MA, US
Justin M. PIERSON - Cambridge MA, US
Muthiah MANOHARAN - Cambridge MA, US
Dale C. GUENTHER - Cambridge MA, US
Ivan ZLATEV - Cambridge MA, US
Christopher S. THEILE - Cambridge MA, US
Vasant R. JADHAV - Cambridge MA, US
Stuart MILSTEIN - Cambridge MA, US
Maja JANAS - Cambridge MA, US
Dhrubajyoti DATTA - Cambridge MA, US
International Classification:
A61K 31/713
A61K 48/00
C12N 15/113
A61P 25/28
Abstract:
One aspect of the present invention relates to a compound comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic monomers, containing one or more lipophilic moieties, conjugated to one or more positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic monomer-conjugated compound.