IL259964B - Adenovirus-associated vectors for the treatment of mucolipodosis type ii - Google Patents

Adenovirus-associated vectors for the treatment of mucolipodosis type ii

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Publication number
IL259964B
IL259964B IL259964A IL25996418A IL259964B IL 259964 B IL259964 B IL 259964B IL 259964 A IL259964 A IL 259964A IL 25996418 A IL25996418 A IL 25996418A IL 259964 B IL259964 B IL 259964B
Authority
IL
Israel
Prior art keywords
aav
raav
gnptab
optionally
nucleic acid
Prior art date
Application number
IL259964A
Other languages
English (en)
Hebrew (he)
Other versions
IL259964A (en
Original Assignee
Genzyme Corp
Samsung Life Public Welfare Foundation
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, Samsung Life Public Welfare Foundation filed Critical Genzyme Corp
Publication of IL259964A publication Critical patent/IL259964A/en
Publication of IL259964B publication Critical patent/IL259964B/en

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    • A—HUMAN NECESSITIES
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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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    • 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
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    • 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
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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
    • A—HUMAN NECESSITIES
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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
    • A61K48/0083—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 administration regime
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • 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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1288—Transferases for other substituted phosphate groups (2.7.8)
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    • C12Y—ENZYMES
    • 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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    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/07—Animals genetically altered by homologous recombination
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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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/00—Animals characterised by purpose
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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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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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • 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
    • 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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  • Bioinformatics & Cheminformatics (AREA)
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  • Virology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurosurgery (AREA)
  • Animal Husbandry (AREA)
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  • Biophysics (AREA)
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  • Diabetes (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
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IL259964A 2015-12-15 2016-12-14 Adenovirus-associated vectors for the treatment of mucolipodosis type ii IL259964B (en)

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 (2)

Publication Number Publication Date
IL259964A IL259964A (en) 2018-07-31
IL259964B true IL259964B (en) 2022-09-01

Family

ID=57737987

Family Applications (1)

Application Number Title Priority Date Filing Date
IL259964A IL259964B (en) 2015-12-15 2016-12-14 Adenovirus-associated vectors for the treatment of mucolipodosis type ii

Country Status (13)

Country Link
US (1) US20200261598A1 (OSRAM)
EP (1) EP3390644B1 (OSRAM)
JP (3) JP7347932B2 (OSRAM)
KR (1) KR102833376B1 (OSRAM)
CN (1) CN108603199A (OSRAM)
AU (2) AU2016372035B2 (OSRAM)
CA (1) CA3008268A1 (OSRAM)
ES (1) ES2991277T3 (OSRAM)
IL (1) IL259964B (OSRAM)
MX (1) MX2018007330A (OSRAM)
RU (1) RU2742612C2 (OSRAM)
SG (2) SG10201912935WA (OSRAM)
WO (1) WO2017106313A1 (OSRAM)

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US11491241B2 (en) 2017-11-07 2022-11-08 The University Of North Carolina At Chapel Hill Optimized AGA genes and expression cassettes and their use
AU2019385506A1 (en) * 2018-11-21 2021-06-03 Takeda Pharmaceutical Company Limited Recombinant viral vectors and nucleic acids for producing the same
WO2021077101A1 (en) 2019-10-18 2021-04-22 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
MX2022009883A (es) * 2020-02-13 2022-10-03 Tenaya Therapeutics Inc Vectores de genoterapia para tratar enfermedades cardíacas.

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DATABASE UNIPROT [ONLINE], RECNAME: FULL=N-ACETYLGLUCOSAMINE-1-PHOSPHOTRANSFERASE SUBUNITS ALPHA/BETA; EC=2.7.8.17 {EC0:00002691PUBMED: 19955174}; ALTNAME: FULL=GLCNAC-1-PHOSPHOTRANSFERASE SUBUNITS ALPHA/BETA; ALTNAME: FULL=STEALTH PROTEIN GNPTAB;ALTNAME: FULL=UDP-N-ACETYLGLUC, 7 March 2006 (2006-03-07) *
GRAY, STEVEN J., ET AL., OPTIMIZING PROMOTERS FOR RECOMBINANT ADENO-ASSOCIATED VIRUS-MEDIATED GENE EXPRESSION IN THE PERIPHERAL AND CENTRAL NERVOUS SYSTEM USING SELF-COMPLEMENTARY VECTORS., 8 April 2011 (2011-04-08) *
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