MX2018007330A - Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. - Google Patents

Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.

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Publication number
MX2018007330A
MX2018007330A MX2018007330A MX2018007330A MX2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A
Authority
MX
Mexico
Prior art keywords
mucolipidosis type
iii
vectors
raav
adeno
Prior art date
Application number
MX2018007330A
Other languages
English (en)
Spanish (es)
Inventor
Yew Nelson
Jin Dong-Kyu
Original Assignee
Genzyme Corp
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.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of MX2018007330A publication Critical patent/MX2018007330A/es

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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    • 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
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    • A61K49/00—Preparations for testing in vivo
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    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/08—Transferases for other substituted phosphate groups (2.7.8)
    • C12Y207/08017—UDP-N-acetylglucosamine--lysosomal-enzyme N-acetylglucosaminephosphotransferase (2.7.8.17)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A—HUMAN NECESSITIES
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    • A01K2217/077—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
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    • 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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MX2018007330A 2015-12-15 2016-12-14 Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. MX2018007330A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562267502P 2015-12-15 2015-12-15
PCT/US2016/066611 WO2017106313A1 (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii

Publications (1)

Publication Number Publication Date
MX2018007330A true MX2018007330A (es) 2019-03-14

Family

ID=57737987

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018007330A MX2018007330A (es) 2015-12-15 2016-12-14 Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.

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US (1) US20200261598A1 (https=)
EP (1) EP3390644B1 (https=)
JP (3) JP7347932B2 (https=)
KR (1) KR102833376B1 (https=)
CN (1) CN108603199A (https=)
AU (2) AU2016372035B2 (https=)
CA (1) CA3008268A1 (https=)
ES (1) ES2991277T3 (https=)
IL (1) IL259964B (https=)
MX (1) MX2018007330A (https=)
RU (1) RU2742612C2 (https=)
SG (2) SG11201804994QA (https=)
WO (1) WO2017106313A1 (https=)

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WO2020106916A1 (en) * 2018-11-21 2020-05-28 Stridebio, Inc. Recombinant viral vectors and nucleic acids for producing the same
CA3158131A1 (en) * 2019-10-18 2021-04-22 Nicolas Sebastien Wein Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
EP4103214B1 (en) * 2020-02-13 2026-04-29 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease

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SG10201912935WA (en) 2020-02-27
AU2023204116A1 (en) 2023-07-13
US20200261598A1 (en) 2020-08-20
WO2017106313A1 (en) 2017-06-22
AU2023204116B2 (en) 2026-03-12
ES2991277T3 (es) 2024-12-03
JP2019500034A (ja) 2019-01-10
AU2016372035B2 (en) 2023-03-30
KR20180098294A (ko) 2018-09-03
IL259964A (en) 2018-07-31
RU2018124448A3 (https=) 2020-08-14
BR112018012015A2 (pt) 2018-12-04
IL259964B (en) 2022-09-01
JP7347932B2 (ja) 2023-09-20
EP3390644A1 (en) 2018-10-24
CN108603199A (zh) 2018-09-28
JP2022046635A (ja) 2022-03-23
KR102833376B1 (ko) 2025-07-11
JP2024056832A (ja) 2024-04-23
CA3008268A1 (en) 2017-06-22
AU2016372035A1 (en) 2018-08-02
EP3390644B1 (en) 2024-07-31
RU2742612C2 (ru) 2021-02-09
SG11201804994QA (en) 2018-07-30
RU2018124448A (ru) 2020-01-17

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