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Title:
TREATMENT OF GENETIC DISEASES CHARACTERIZED BY UNSTABLE mRNAs
Document Type and Number:
WIPO Patent Application WO/2021/059270
Kind Code:
A3
Abstract:
Methods of treating a disease characterized by mRNA instability or nonsense-mediated decay of an mRNA of a disease-associated gene in a subject by administering a pharmaceutical composition comprising at least one agent that decreases FTO expression, function or both are provided. Kits and pharmaceutical compositions comprising an agent that decreases FTO expression, function or both and a read-through promoting agent are also provided, as are methods of determining suitability of a subject to be treated with an agent that decreases FTO expression, function or both.

Inventors:
KARNI ROTEM (IL)
AMAR-SCHWARTZ ADI (IL)
Application Number:
PCT/IL2020/051038
Publication Date:
May 06, 2021
Filing Date:
September 23, 2020
Export Citation:
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Assignee:
YISSUM RES DEV CO OF HEBREW UNIV JERUSALEM LTD (IL)
International Classes:
A61K31/196; A61K9/00; A61K31/4245; A61K31/436; A61K31/4418; A61K31/455; A61K31/55; A61K31/706; A61K45/06; A61P35/00; G01N33/00
Domestic Patent References:
WO2018169994A12018-09-20
WO2009045443A22009-04-09
WO2019148282A12019-08-08
WO2019143743A12019-07-25
Other References:
PUSHKAR MALAKAR ET AL: "Long Noncoding RNA MALAT1 Regulates Cancer Glucose Metabolism by Enhancing mTOR-Mediated Translation of TCF7L2", CANCER RESEARCH, vol. 79, no. 10, 15 May 2019 (2019-05-15), US, pages 2480 - 2493, XP055766153, ISSN: 0008-5472, DOI: 10.1158/0008-5472.CAN-18-1432
Attorney, Agent or Firm:
KESTEN, Dov et al. (IL)
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