IP Library › Granted Patent US 11,674,140
Granted Patent B2
US 11,674,140 · App. 16/643,772 · Granted Jun 13, 2023

Compositions and methods for treating facioscapulohumeral dystrophy

Inventors: Angela Lek (Boston, MA); Louis M. Kunkel (Boston, MA)
Assignee: THE CHILDREN'S MEDICAL CENTER CORPORATION
C12N15/113A61K31/366A61K31/395A61K31/436A61K31/5377A61P21/00C12N15/1093C12N15/1137C12N2310/3233
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Quick Facts
Patent No.
US 11,674,140
App. No.
16/643,772
Granted
Jun 13, 2023
Kind
B2
Abstract

Compositions and methods for treating FSHD and for identifying agents useful for the treatment of FSHD.

Claims (1)

1. A method for suppressing DUX4 toxicity in a cell, the method comprising contacting a cell overexpressing DUX4 with an agent that reduces the expression or activity of a hypoxia response pathway protein selected from the group consisting of hypoxia-inducible factor 1-alpha (HIF1A), hypoxia-inducible factor 1-beta (HIF1B), aryl hydrocarbon receptor nuclear translocator (ARNT), CREB binding protein (CREBBP), CBP, and zinc finger CCHC-type containing 14 (ZCCHC14), wherein the agent is Herbimycin A, Herceptin, Iressa, calphostin C, wortmannin, LY294002, PD98059, rapamycin, diphenylene iodonium, mannoheptulose, 4-Chloro-3-(trifluoromethyl)phenyl isocyanate (CAS 327-78-6), Sterigmatocystin, Chetomin, Cryptotanshione, EF-24, FM19G11, and PX 12.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 23, 2020
From: LEK, ANGELA; KUNKEL, LOUIS M.
To: THE CHILDREN'S MEDICAL CENTER CORPORATION
Reel/Frame 054148/0265 →
Continuity (2)
Provisional Application 62555599 · Sep 7, 2017
Related Publication 20210163931A1 · Jun 3, 2021