MX2017002900A - Terapia genica de globina para el tratamiento de hemoglobinopatias. - Google Patents

Terapia genica de globina para el tratamiento de hemoglobinopatias.

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Publication number
MX2017002900A
MX2017002900A MX2017002900A MX2017002900A MX2017002900A MX 2017002900 A MX2017002900 A MX 2017002900A MX 2017002900 A MX2017002900 A MX 2017002900A MX 2017002900 A MX2017002900 A MX 2017002900A MX 2017002900 A MX2017002900 A MX 2017002900A
Authority
MX
Mexico
Prior art keywords
hemoglobinopatias
globine
genetic
therapy
treatment
Prior art date
Application number
MX2017002900A
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English (en)
Other versions
MX382337B (es
Inventor
Michel Sadelain
Riviere Isabelle
MANSILLA-SOTO Jorge
Wang Xiuyan
Stamatoyannopoulos George
STAMATOYANNOPOULOS John
Liu Mingdong
Original Assignee
Memorial Sloan Kettering Cancer Center
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Application filed by Memorial Sloan Kettering Cancer Center filed Critical Memorial Sloan Kettering Cancer Center
Publication of MX2017002900A publication Critical patent/MX2017002900A/es
Publication of MX382337B publication Critical patent/MX382337B/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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector 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 That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

La materia en cuestión descrita en la presente invención proporciona cartuchos de expresión que permiten la expresi6n de un gen de globina o una parte funcional de los mismos, vectores que lo comprenden y células transducidas con dichos cartuchos y vectores de expresión; la materia objeto de la presente descripción proporciona además métodos para tratar una hemoglobinopatía en un sujeto que comprende administrar una cantidad eficaz de tales células transducidas al sujeto.
MX2017002900A 2014-09-04 2015-09-04 Terapia génica de globina y uso de la misma para el tratamiento de hemoglobinopatias. MX382337B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462045997P 2014-09-04 2014-09-04
PCT/US2015/048698 WO2016037138A1 (en) 2014-09-04 2015-09-04 Global gene therapy for treating hemoglobinopathies

Publications (2)

Publication Number Publication Date
MX2017002900A true MX2017002900A (es) 2017-10-24
MX382337B MX382337B (es) 2025-03-13

Family

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MX2017002900A MX382337B (es) 2014-09-04 2015-09-04 Terapia génica de globina y uso de la misma para el tratamiento de hemoglobinopatias.

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US (2) US11717579B2 (es)
EP (3) EP3620521B1 (es)
JP (3) JP6710680B2 (es)
KR (2) KR102500531B1 (es)
CN (3) CN114457076A (es)
AU (2) AU2015311681B2 (es)
BR (1) BR112017004349A2 (es)
CA (1) CA2960209C (es)
ES (1) ES2746529T3 (es)
MX (1) MX382337B (es)
WO (1) WO2016037138A1 (es)

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