MX2022003116A - Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. - Google Patents

Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.

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Publication number
MX2022003116A
MX2022003116A MX2022003116A MX2022003116A MX2022003116A MX 2022003116 A MX2022003116 A MX 2022003116A MX 2022003116 A MX2022003116 A MX 2022003116A MX 2022003116 A MX2022003116 A MX 2022003116A MX 2022003116 A MX2022003116 A MX 2022003116A
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Mexico
Prior art keywords
organs
tissues
cells
gene transfer
methods
Prior art date
Application number
MX2022003116A
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English (en)
Inventor
Katherine A High
Xavier Anguela
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Childrens Hospital Philadelphia
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Publication date
Application filed by Childrens Hospital Philadelphia filed Critical Childrens Hospital Philadelphia
Publication of MX2022003116A publication Critical patent/MX2022003116A/es

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    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/36—Blood coagulation or fibrinolysis factors
    • 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
    • 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
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    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
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    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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    • C12N2750/14011—Parvoviridae
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    • 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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Abstract

La invención se refiere a una secuencia que codifica el Factor IX modificado, el casete de expresión, los vectores tales como vectores virales (por ejemplo, lenti- o adenoasociados virales), y métodos y usos de transferencia genética. En particular, se refiere a dirigir el ácido nucleico del Factor IX a las células, los tejidos u órganos para la expresión (transcripción) del Factor IX.
MX2022003116A 2015-06-23 2018-01-08 Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. MX2022003116A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562183599P 2015-06-23 2015-06-23
US201662315453P 2016-03-30 2016-03-30
US201662338315P 2016-05-18 2016-05-18
US201662348781P 2016-06-10 2016-06-10
US201662349572P 2016-06-13 2016-06-13

Publications (1)

Publication Number Publication Date
MX2022003116A true MX2022003116A (es) 2022-04-11

Family

ID=57586246

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MX2018000307A MX2018000307A (es) 2015-06-23 2016-06-23 Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
MX2022003116A MX2022003116A (es) 2015-06-23 2018-01-08 Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.

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US (5) US10799566B2 (es)
EP (1) EP3313991A4 (es)
JP (3) JP6831779B2 (es)
KR (2) KR102178322B1 (es)
CN (1) CN108138159A (es)
AU (2) AU2016282781A1 (es)
BR (1) BR112017027956A2 (es)
CA (1) CA2990193A1 (es)
CO (1) CO2018000134A2 (es)
HK (1) HK1252351A1 (es)
IL (2) IL256517B1 (es)
MX (2) MX2018000307A (es)
PE (1) PE20180675A1 (es)
PH (1) PH12017502424A1 (es)
SA (1) SA517390590B1 (es)
SG (1) SG10202100131WA (es)
WO (1) WO2016210170A1 (es)

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