MX2022002652A - Metodos para restaurar la funcion lisosomal de celulas epiteliales de pigmento retiniano mediante activacion de tfeb. - Google Patents

Metodos para restaurar la funcion lisosomal de celulas epiteliales de pigmento retiniano mediante activacion de tfeb.

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Publication number
MX2022002652A
MX2022002652A MX2022002652A MX2022002652A MX2022002652A MX 2022002652 A MX2022002652 A MX 2022002652A MX 2022002652 A MX2022002652 A MX 2022002652A MX 2022002652 A MX2022002652 A MX 2022002652A MX 2022002652 A MX2022002652 A MX 2022002652A
Authority
MX
Mexico
Prior art keywords
tfeb
pigment epithelial
retinal pigment
activation
methods
Prior art date
Application number
MX2022002652A
Other languages
English (en)
Inventor
Debasish Sinha
Leah Caroline Thomas Byrne
Nadezda Anatolyena Stepicheva
Sayan Ghosh
Stacey Hose
Original Assignee
Univ Pittsburgh Commonwealth Sys Higher Education
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 Univ Pittsburgh Commonwealth Sys Higher Education filed Critical Univ Pittsburgh Commonwealth Sys Higher Education
Publication of MX2022002652A publication Critical patent/MX2022002652A/es

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    • 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
    • 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
    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • 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
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/07—Animals genetically altered by homologous recombination
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K9/0012—Galenical forms characterised by the site of application
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/60—Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Environmental Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Animal Husbandry (AREA)
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  • Ophthalmology & Optometry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Virology (AREA)
  • Hospice & Palliative Care (AREA)

Abstract

Se proporciona un método para restaurar la función lisosomal de células epiteliales de pigmento retiniano (RPE) y un método para prevenir y/o tratar degeneración macular relacionada con la edad (AMD), degeneración macular retiniana de Stargardt, enfermedad neurodegenerativa, o retinopatía diabética en un sujeto. Los métodos comprenden administrar (i) un ácido nucleico que codifica para un polipéptido que comprende una forma constitutivamente activa del factor de transcripción EB (TFEB) o (ii) el polipéptido, a un sujeto en necesidad del mismo. También se proporcionan polipéptidos asociados, ácidos nucleicos, vectores, y composiciones de los mismos.
MX2022002652A 2019-09-09 2020-03-06 Metodos para restaurar la funcion lisosomal de celulas epiteliales de pigmento retiniano mediante activacion de tfeb. MX2022002652A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962897800P 2019-09-09 2019-09-09
PCT/US2020/021540 WO2021050102A1 (en) 2019-09-09 2020-03-06 Methods of restoring lysosomal function of retinal pigment epithelial cells by activation of tfeb

Publications (1)

Publication Number Publication Date
MX2022002652A true MX2022002652A (es) 2022-04-06

Family

ID=74867139

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022002652A MX2022002652A (es) 2019-09-09 2020-03-06 Metodos para restaurar la funcion lisosomal de celulas epiteliales de pigmento retiniano mediante activacion de tfeb.

Country Status (12)

Country Link
US (1) US20220313722A1 (es)
EP (1) EP4028025A4 (es)
JP (1) JP2022547158A (es)
KR (1) KR20220062577A (es)
CN (1) CN114828857A (es)
AU (1) AU2020344449A1 (es)
BR (1) BR112022003508A2 (es)
CA (1) CA3153963A1 (es)
CO (1) CO2022003930A2 (es)
IL (1) IL291077A (es)
MX (1) MX2022002652A (es)
WO (1) WO2021050102A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202405180A (zh) * 2022-03-30 2024-02-01 高等教育聯邦系統 匹茲堡大學 用於將核酸遞送至視網膜細胞的腺相關病毒載體

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2196496A1 (en) * 1997-01-31 1998-07-31 Stephen William Watson Michnick Protein fragment complementation assay for the detection of protein-protein interactions
WO2009114729A2 (en) * 2008-03-14 2009-09-17 Irm Llc Compounds, compositions and methods for treating lysosomal storage diseases and disorders
EP2218458A1 (en) * 2009-02-13 2010-08-18 Fondazione Telethon Molecules able to modulate the expression of at least a gene involved in degradative pathways and uses thereof
WO2012120044A1 (en) * 2011-03-07 2012-09-13 Fondazione Telethon Tfeb variants and uses thereof
WO2012177968A1 (en) * 2011-06-22 2012-12-27 The Schepens Eye Research Institute, Inc. A scaffold for subretinal cell transplantation and drug delivery
WO2013156625A1 (en) * 2012-04-19 2013-10-24 Fondazione Telethon Tfeb gene therapy of alpha-1-antitrypsin deficiency
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
JP2017536363A (ja) * 2014-11-19 2017-12-07 ラッシュ・ユニバーシティ・メディカル・センター リソソーム蓄積症治療のための組成物及び方法
CN106619600B (zh) * 2016-03-28 2019-10-18 中国科学院遗传与发育生物学研究所 巨大戟醇及其衍生物在增强溶酶体生成中的应用
EP3445451B1 (en) * 2016-04-21 2021-08-18 Baylor College of Medicine Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction

Also Published As

Publication number Publication date
IL291077A (en) 2022-05-01
EP4028025A4 (en) 2023-07-19
BR112022003508A2 (pt) 2022-06-28
EP4028025A1 (en) 2022-07-20
KR20220062577A (ko) 2022-05-17
CA3153963A1 (en) 2021-03-18
CO2022003930A2 (es) 2022-04-19
WO2021050102A1 (en) 2021-03-18
CN114828857A (zh) 2022-07-29
JP2022547158A (ja) 2022-11-10
US20220313722A1 (en) 2022-10-06
AU2020344449A1 (en) 2022-04-07

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