BR112022003508A2 - Métodos para restaurar a função lisossômica de células epiteliais de pigmento da retina por ativação de tfeb - Google Patents

Métodos para restaurar a função lisossômica de células epiteliais de pigmento da retina por ativação de tfeb

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Publication number
BR112022003508A2
BR112022003508A2 BR112022003508A BR112022003508A BR112022003508A2 BR 112022003508 A2 BR112022003508 A2 BR 112022003508A2 BR 112022003508 A BR112022003508 A BR 112022003508A BR 112022003508 A BR112022003508 A BR 112022003508A BR 112022003508 A2 BR112022003508 A2 BR 112022003508A2
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Brazil
Prior art keywords
epithelial cells
pigment epithelial
retinal pigment
methods
tfeb
Prior art date
Application number
BR112022003508A
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English (en)
Inventor
Sinha Debasish
Caroline Thomas Byrne Leah
Anatolyena Stepicheva Nadezda
Ghosh Sayan
Hose Stacey
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Univ Pittsburgh Commonwealth Sys Higher Education
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Publication date
Application filed by Univ Pittsburgh Commonwealth Sys Higher Education filed Critical Univ Pittsburgh Commonwealth Sys Higher Education
Publication of BR112022003508A2 publication Critical patent/BR112022003508A2/pt

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Abstract

MÉTODOS PARA RESTAURAR A FUNÇÃO LISOSSÔNICA DE CÉLULAS EPITELIAIS DE PIGMENTO DA RETINA POR ATIVAÇÃO DE TFEB. A presente invenção refere-se a um método para restaurar a função lisossômica de células epiteliais de pigmento da retina (RPE) e um método para prevenir e/ou tratar a degeneração macular relacionada à idade (DMRI), degeneração macular da retina de Stargardt, doença neurodegenerativa ou retinopatia diabética em um indivíduo são fornecidos. Os métodos compreendem a administração de (i) um ácido nucleico que codifica um polipeptídeo compreendendo uma forma constitutivamente ativa do fator de transcrição EB (TFEB) ou (ii) o polipeptídeo a um indivíduo em necessidade do mesmo. Polipeptídeos associados, ácidos nucleicos, vetores e composições dos mesmos também são fornecidos.
BR112022003508A 2019-09-09 2020-03-06 Métodos para restaurar a função lisossômica de células epiteliais de pigmento da retina por ativação de tfeb BR112022003508A2 (pt)

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
BR112022003508A2 true BR112022003508A2 (pt) 2022-06-28

Family

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BR112022003508A BR112022003508A2 (pt) 2019-09-09 2020-03-06 Métodos para restaurar a função lisossômica de células epiteliais de pigmento da retina por ativação de tfeb

Country Status (12)

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

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* 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

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Publication number Publication date
IL291077A (en) 2022-05-01
EP4028025A4 (en) 2023-07-19
EP4028025A1 (en) 2022-07-20
MX2022002652A (es) 2022-04-06
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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