WO2019118727A3 - Rescue of the pathology of lrrk2 on lysosmes with snx25 or snx27 - Google Patents

Rescue of the pathology of lrrk2 on lysosmes with snx25 or snx27 Download PDF

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Publication number
WO2019118727A3
WO2019118727A3 PCT/US2018/065451 US2018065451W WO2019118727A3 WO 2019118727 A3 WO2019118727 A3 WO 2019118727A3 US 2018065451 W US2018065451 W US 2018065451W WO 2019118727 A3 WO2019118727 A3 WO 2019118727A3
Authority
WO
WIPO (PCT)
Prior art keywords
snx25
snx27
lrrk2
lysosmes
rescue
Prior art date
Application number
PCT/US2018/065451
Other languages
French (fr)
Other versions
WO2019118727A2 (en
Inventor
Asa Abeliovich
Original Assignee
The Trustees Of Columbia University In The City Of New York
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by The Trustees Of Columbia University In The City Of New York filed Critical The Trustees Of Columbia University In The City Of New York
Publication of WO2019118727A2 publication Critical patent/WO2019118727A2/en
Publication of WO2019118727A3 publication Critical patent/WO2019118727A3/en

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5014—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics for testing toxicity
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/573—Immunoassay; Biospecific binding assay; Materials therefor for enzymes or isoenzymes
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/58—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving labelled substances
    • G01N33/582—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving labelled substances with fluorescent label
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/2835—Movement disorders, e.g. Parkinson, Huntington, Tourette

Abstract

The present invention relates to methods of inhibiting, treating, or preventing LRRK2 kinase inhibitor lysosomal toxicity in a subject in need thereof comprising administering to the subject a therapeutically effective amount of a nucleic acid encoding SNX25, a nucleic acid encoding SNX27, a SNX25 polypeptide, a SNX27 polypeptide, a SNX25 activator or agonist, a SNX27 activator or agonist, or a combination thereof.
PCT/US2018/065451 2017-12-14 2018-12-13 Rescue of the pathology of lrrk2 on lysosmes with snx25 or snx27 WO2019118727A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762598654P 2017-12-14 2017-12-14
US62/598,654 2017-12-14

Publications (2)

Publication Number Publication Date
WO2019118727A2 WO2019118727A2 (en) 2019-06-20
WO2019118727A3 true WO2019118727A3 (en) 2019-07-25

Family

ID=66819754

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2018/065451 WO2019118727A2 (en) 2017-12-14 2018-12-13 Rescue of the pathology of lrrk2 on lysosmes with snx25 or snx27

Country Status (1)

Country Link
WO (1) WO2019118727A2 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3769768A1 (en) * 2019-07-23 2021-01-27 Consejo Superior de Investigaciones Cientificas (CSIC) Compounds useful for the treatment of parkinson s disease
CN113999847A (en) * 2021-10-11 2022-02-01 中山大学附属第六医院 sgRNA of specific target LRRK2 gene, lentivirus transfection vector and application thereof
CN117625626B (en) * 2024-01-25 2024-04-16 中国农业科学院植物保护研究所 Application of RNAi in improving effect of bacillus thuringiensis insecticidal protein in preventing and controlling chilo suppressalis or spodoptera frugiperda

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090004112A1 (en) * 2006-04-21 2009-01-01 The Trustees Of Columbia University In The City Of New York Methods for the treatment of neurodegenerative diseases using nmda receptor glycine site antagonists
US20160184454A1 (en) * 2013-01-11 2016-06-30 Asa Abeliovich Rab7l1 interacts with lrrk2 to modify intraneuronal protein sorting and parkinson's disease risk

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090004112A1 (en) * 2006-04-21 2009-01-01 The Trustees Of Columbia University In The City Of New York Methods for the treatment of neurodegenerative diseases using nmda receptor glycine site antagonists
US20160184454A1 (en) * 2013-01-11 2016-06-30 Asa Abeliovich Rab7l1 interacts with lrrk2 to modify intraneuronal protein sorting and parkinson's disease risk

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ATASHRAZM ET AL.: "LRRK2 inhibitors and their potential in the treatment of Parkinson's disease: current perspectives", CLINICAL PHARMACOLOGY: ADVANCES AND APPLICATIONS, vol. 8, 20 October 2016 (2016-10-20), pages 177 - 189, XP055628072 *
WANG ET AL.: "Loss of sorting nexin 27 contributes to excitatory synaptic dysfunction by modulating glutamate receptor recycling in Down's syndrome", NATURE MEDICINE, vol. 19, no. 4, April 2013 (2013-04-01), pages 473 - 480, XP055628074 *

Also Published As

Publication number Publication date
WO2019118727A2 (en) 2019-06-20

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