Title:
T-TYPE CALCIUM CHANNEL ENHANCER FOR TREATING TAF1 ASSOCIATED NEUROLOGICAL DEFECTS
Document Type and Number:
WIPO Patent Application WO/2021/252950
Kind Code:
A3
Abstract:
Dysregulation of TAF1 function by various mechanisms can lead to disease in the central nervous system, such as the TAF1 Intellectual Disability (ID) Syndrome which currently has no therapeutic treatments. The present invention indicates that a novel T-type calcium channel enhancer, such as SAK3, has disease-modifying effects in animal models of TAF1 editing. In addition, the present invention provides insights into the molecular mechanism by which SAK3 exerts in pharmacologic effects. Moreover, the present findings imply that the T-Type voltage-gated calcium channels are novel molecular targets to develop therapeutics to treat TAF1 ID syndrome and that SAK3 is an attractive drug candidate to treat TAF1 associated neurologic disorders.
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Inventors:
NELSON MARK (US)
KHANNA RAJESH (US)
UDAIYAPPAN JANAKIRAMAN (US)
SHANKARA RAMAN DHANALAKSHMI (GB)
KHANNA RAJESH (US)
UDAIYAPPAN JANAKIRAMAN (US)
SHANKARA RAMAN DHANALAKSHMI (GB)
Application Number:
PCT/US2021/037085
Publication Date:
January 27, 2022
Filing Date:
June 11, 2021
Export Citation:
Assignee:
UNIV ARIZONA (US)
International Classes:
A61P25/00; A61K31/437; A61K31/438; A61P25/28
Foreign References:
| US20150045385A1 | 2015-02-12 |
Other References:
JANAKIRAMAN UDAIYAPPAN; YU JIE; MOUTAL AUBIN; CHINNASAMY DHANALAKSHMI; BOINON LISA; BATCHELOR SHELBY N.; ANANDHAN ANNADURI; KHANNA: "TAF1-gene editing alters the morphology and function of the cerebellum and cerebral cortex", NEUROBIOLOGY OF DISEASE, ELSEVIER, AMSTERDAM, NL, vol. 132, 22 July 2019 (2019-07-22), AMSTERDAM, NL , XP085887629, ISSN: 0969-9961, DOI: 10.1016/j.nbd.2019.104539
USUI NORIYOSHI; CO MARISSA; HARPER MATTHEW; RIEGER MICHAEL A.; DOUGHERTY JOSEPH D.; KONOPKA GENEVIEVE: "Sumoylation of FOXP2 Regulates Motor Function and Vocal Communication Through Purkinje Cell Development", BIOLOGICAL PSYCHIATRY, ELSEVIER, AMSTERDAM, NL, vol. 81, no. 3, 13 February 2016 (2016-02-13), AMSTERDAM, NL, pages 220 - 230, XP029862642, ISSN: 0006-3223, DOI: 10.1016/j.biopsych.2016.02.008
USUI NORIYOSHI; CO MARISSA; HARPER MATTHEW; RIEGER MICHAEL A.; DOUGHERTY JOSEPH D.; KONOPKA GENEVIEVE: "Sumoylation of FOXP2 Regulates Motor Function and Vocal Communication Through Purkinje Cell Development", BIOLOGICAL PSYCHIATRY, ELSEVIER, AMSTERDAM, NL, vol. 81, no. 3, 13 February 2016 (2016-02-13), AMSTERDAM, NL, pages 220 - 230, XP029862642, ISSN: 0006-3223, DOI: 10.1016/j.biopsych.2016.02.008
Attorney, Agent or Firm:
NGUYEN, Quan (US)
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