CL2020002704A1 - Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial - Google Patents

Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial

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Publication number
CL2020002704A1
CL2020002704A1 CL2020002704A CL2020002704A CL2020002704A1 CL 2020002704 A1 CL2020002704 A1 CL 2020002704A1 CL 2020002704 A CL2020002704 A CL 2020002704A CL 2020002704 A CL2020002704 A CL 2020002704A CL 2020002704 A1 CL2020002704 A1 CL 2020002704A1
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CL
Chile
Prior art keywords
gene therapy
mitochondrial dna
treatment
dna depletion
involve
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CL2020002704A
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English (en)
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Michio Hirano
Hasan O Akman
Carlos Lopez-Gomez
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Univ Columbia
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Publication of CL2020002704A1 publication Critical patent/CL2020002704A1/es

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    • 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
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
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    • A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • A61K31/7072—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
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Abstract

La invención se refiere en general, a un método de tratamiento para una enfermedad genética humana, tal como enfermedades caracterizadas por combinaciones desequilibradas de nucleótidos, por ejemplo, síndrome de agotamiento de ADN mitocondriales, y más específicamente, deficiencia de timidina cinasa 2 (TK2), usando terapia de gen. La terapia de gen puede involucrar la administración de uno o más constructos, tal como un vector viral, que contiene un ácido nucleico que codifica a una proteína funcional. La proteína funcional puede corresponder a un gen nuclear. Para tratamiento de deficiencia de TK2, la terapia de gen puede involucrar la administración de uno o más constructos, tal como un vector viral, que contiene un ácido nucleico que codifica a una enzima de proteína funcional TK2. El tratamiento puede involucrar también la administración de terapia farmacológica en conjunto con la terapia de gen. Los protocolos de tratamiento de la descripción, tal como aquellos involucran terapia de gen solos o en combinación con terapia farmacológica, pueden ser usados para tratar, prevenir, y/o curar varios otros trastornos de combinaciones desequilibradas de nucleósidos, especialmente aquellos encontrados en el síndrome de agotamiento de ADN mitocondrial.
CL2020002704A 2018-04-18 2020-10-19 Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial CL2020002704A1 (es)

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US201862659281P 2018-04-18 2018-04-18

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CL2020002704A1 true CL2020002704A1 (es) 2021-01-29

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US (1) US12419970B2 (es)
EP (1) EP3781671A4 (es)
JP (1) JP7769355B2 (es)
KR (1) KR20210009317A (es)
CN (1) CN112312929A (es)
AU (1) AU2019255745A1 (es)
BR (1) BR112020021324A2 (es)
CA (1) CA3097392A1 (es)
CL (1) CL2020002704A1 (es)
CO (1) CO2020014193A2 (es)
IL (1) IL278130B2 (es)
MX (1) MX2020010994A (es)
WO (1) WO2019204593A1 (es)

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KR102962433B1 (ko) * 2023-06-30 2026-05-08 사회복지법인 삼성생명공익재단 Hydra NDi1가 탑재된 재조합 아데노부속바이러스 벡터를 포함하는, 미토콘드리아 복합체 1 이상질환의 예방, 개선 또는 치료용 조성물
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EP2009990B1 (en) * 2006-04-07 2016-09-21 The Board of Regents of The University of Texas System Methods and compositions related to adenoassociated virus-phage particles
FR3004463A1 (fr) * 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
US10292996B2 (en) * 2015-03-26 2019-05-21 The Trustees Of Columbia University In The City Of New York Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome
WO2016205671A1 (en) 2015-06-17 2016-12-22 The Trustees Of Columbia University In The City Of New York Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes
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