CO2020014193A2 - Terapia genética para las enfermedades causadas por piscinas de nucleótidos desequilibradas, incluyendo los síndromes de depleción del adn mitocondrial - Google Patents

Terapia genética para las enfermedades causadas por piscinas de nucleótidos desequilibradas, incluyendo los síndromes de depleción del adn mitocondrial

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Publication number
CO2020014193A2
CO2020014193A2 CONC2020/0014193A CO2020014193A CO2020014193A2 CO 2020014193 A2 CO2020014193 A2 CO 2020014193A2 CO 2020014193 A CO2020014193 A CO 2020014193A CO 2020014193 A2 CO2020014193 A2 CO 2020014193A2
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Colombia
Prior art keywords
gene therapy
mitochondrial dna
treatment
dna depletion
involve
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CONC2020/0014193A
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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 date
Application filed by Univ Columbia filed Critical Univ Columbia
Publication of CO2020014193A2 publication Critical patent/CO2020014193A2/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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    • A61K31/7052—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
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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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    • C12N9/1205—Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein kinases
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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.
CONC2020/0014193A 2018-04-18 2020-11-17 Terapia genética para las enfermedades causadas por piscinas de nucleótidos desequilibradas, incluyendo los síndromes de depleción del adn mitocondrial CO2020014193A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862659281P 2018-04-18 2018-04-18
PCT/US2019/028108 WO2019204593A1 (en) 2018-04-18 2019-04-18 Gene therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes

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CN (1) CN112312929A (es)
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CA (1) CA3097392A1 (es)
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AU2019255745A1 (en) 2020-11-19
WO2019204593A1 (en) 2019-10-24
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JP2021522173A (ja) 2021-08-30
MX2020010994A (es) 2021-01-08
US20210100917A1 (en) 2021-04-08
BR112020021324A2 (pt) 2021-01-19
KR20210009317A (ko) 2021-01-26
CL2020002704A1 (es) 2021-01-29
EP3781671A4 (en) 2022-01-26
CN112312929A (zh) 2021-02-02

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