MX2023001615A - Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav. - Google Patents

Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav.

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Publication number
MX2023001615A
MX2023001615A MX2023001615A MX2023001615A MX2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A
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Mexico
Prior art keywords
pkp2
plakophilin
gene therapy
vector
promoter
Prior art date
Application number
MX2023001615A
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English (en)
Inventor
Raj Prabhakar
David Ricks
Christopher Dean Herzog
Chester Bittencort Sacramento
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Spacecraft Seven Llc
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Publication date
Application filed by Spacecraft Seven Llc filed Critical Spacecraft Seven Llc
Publication of MX2023001615A publication Critical patent/MX2023001615A/es

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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • AHUMAN NECESSITIES
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

En la presente se proporciona una genoterapia para PKP2 (Placofilina-2), por ejemplo, mediante el uso de un vector de virus adenoasociado (AAV). El promotor del vector puede ser un promotor MHCK7 o un promotor de troponina cardíaca T (HTNNT2). La cápside puede ser una cápside de AAV9 o AAVrh74 o variante funcional de esta. Pueden usarse otros promotores o cápsides. Se proporcionan además métodos de tratamiento, tales como por administración intravenosa, intracoronaria, intracarótida o intracardíaca del vector de rAAV, y otras composiciones y métodos.
MX2023001615A 2020-08-07 2021-08-09 Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav. MX2023001615A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063063032P 2020-08-07 2020-08-07
PCT/US2021/045220 WO2022032226A1 (en) 2020-08-07 2021-08-09 Plakophilin-2 (pkp2) gene therapy using aav vector

Publications (1)

Publication Number Publication Date
MX2023001615A true MX2023001615A (es) 2023-03-08

Family

ID=80118582

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023001615A MX2023001615A (es) 2020-08-07 2021-08-09 Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav.

Country Status (11)

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US (2) US11883506B2 (es)
EP (1) EP4192487A1 (es)
JP (1) JP2023538519A (es)
KR (1) KR20230043869A (es)
CN (1) CN116194154A (es)
AU (1) AU2021320902A1 (es)
BR (1) BR112023001852A2 (es)
CA (1) CA3185267A1 (es)
IL (1) IL300263A (es)
MX (1) MX2023001615A (es)
WO (1) WO2022032226A1 (es)

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