Title:
SYSTEMIC DELIVERY OF MYOSTATIN SHORT INTERFERING NUCLEIC ACIDS (siNA) CONJUGATED TO LIPOPHILIC MOIETY
Document Type and Number:
Japanese Patent JP2020114821
Kind Code:
A
Abstract:
To provide methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, where the small nucleic acid molecules are introduced to a subject by systemic administration.SOLUTION: The invention relates to methods comprising the in vivo delivery of short interfering nucleic acid (siNA) molecules that target a myostatin gene expressed by a subject, where the siNA molecule is conjugated to a lipophilic moiety, such as cholesterol. The myostatin siNA conjugates that are delivered as per the methods disclosed are useful to modulate the in vivo expression of myostatin, increase muscle mass and/or enhance muscle performance. Use of the disclosed methods provides methods for treating musculoskeletal diseases or disorders and/or diseases or disorders that result in conditions in which muscle is adversely affected.SELECTED DRAWING: None
Inventors:
SEPP-LORENZINO LAURA
VASANT R JADHAV
DUNCAN BROWN
MARIJA TADIN-STRAPPS
TAYEBA KHAN
WALTER RICHARD STRAPPS
VASANT R JADHAV
DUNCAN BROWN
MARIJA TADIN-STRAPPS
TAYEBA KHAN
WALTER RICHARD STRAPPS
Application Number:
JP2020036735A
Publication Date:
July 30, 2020
Filing Date:
March 04, 2020
Export Citation:
Assignee:
SIRNA THERAPEUTICS INC
International Classes:
A61K31/713; A61K47/54; A61K48/00; A61P21/00; A61P21/06; C12N15/113
Attorney, Agent or Firm:
Michiko Matsutani
Sakurai Yoko
Keiji Sakata
Sakurai Yoko
Keiji Sakata