Title:
COMBINATION OF RETROMER PHARMACOLOGICAL CHAPERONES AND EXOGENOUS RETROMER FOR TREATMENT OF ALZHEIMER'S DISEASE AND OTHER NEURODEGENERATIVE DISEASES
Document Type and Number:
WIPO Patent Application WO/2021/163681
Kind Code:
A3
Abstract:
The present disclosure relates to methods and compositions for elevating and stabilizing retromer for treating and/or preventing Alzheimer's disease and other neurodegenerative diseases or disorders. Additionally, the disclosure relates to gene therapy combined with a pharmacological retromer chaperone therapy for treating and/or preventing Alzheimer's disease (AD), and other neurodegenerative diseases or disorders such as Parkinson's Disease (PD), amyotrophic lateral sclerosis (ALS), neuronal ceroid lipofuscinosis (NCL), and transmissible spongiform encephalopathies (TSEs or prion disease), multiple system atrophy (MSA), as well as tauopathies such as progressive supranuclear palsy (PSP), frontotemporal lobar dementia linked to chromosome 17q21-22 and its subtypes (FTLD- 17/FTLD -Tau), and chronic traumatic encephalopathy (CTE).
Inventors:
SMALL SCOTT A (US)
PETSKO GREGORY A (US)
PETSKO GREGORY A (US)
Application Number:
PCT/US2021/018167
Publication Date:
March 10, 2022
Filing Date:
February 16, 2021
Export Citation:
Assignee:
UNIV COLUMBIA (US)
International Classes:
A61K48/00; A61P25/28; C12N15/09; C12N15/86
Domestic Patent References:
| WO2019070894A1 | 2019-04-11 |
Foreign References:
| US20160250182A1 | 2016-09-01 | |||
| US5972653A | 1999-10-26 | |||
| US6004778A | 1999-12-21 | |||
| US20080214482A1 | 2008-09-04 |
Other References:
HIOKI: "Efficient gene transduction of neurons by lentivirus with enhanced neuron-specific promoters", GENE THERAPY, vol. 14, 15 March 2007 (2007-03-15), pages 872 - 882, XP055062940, DOI: 10.1038/sj.gt.3302924
Attorney, Agent or Firm:
CAWLEY, Thomas A., Jr. (US)
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