JP2022525136A - パーキンソン病を処置するための遺伝子治療組成物および方法 - Google Patents

パーキンソン病を処置するための遺伝子治療組成物および方法 Download PDF

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JP2022525136A
JP2022525136A JP2021555083A JP2021555083A JP2022525136A JP 2022525136 A JP2022525136 A JP 2022525136A JP 2021555083 A JP2021555083 A JP 2021555083A JP 2021555083 A JP2021555083 A JP 2021555083A JP 2022525136 A JP2022525136 A JP 2022525136A
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nucleotide sequence
vector
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sequence encoding
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JP2022525136A5 (enExample
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ギャビン コーコラン，
ミシェル ストーン，
キリアコス ミトロファヌス，
ハンナ スチュワート，
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サイオ ジーン セラピーズ インコーポレイテッド
オックスフォード バイオメディカ （ユーケー） リミテッド
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
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    • A—HUMAN NECESSITIES
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    • A61P25/16—Anti-Parkinson drugs
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    • C12N15/09—Recombinant DNA-technology
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    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16002—Tyrosine 3-monooxygenase (1.14.16.2)
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    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04016—GTP cyclohydrolase I (3.5.4.16)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01028—Aromatic-L-amino-acid decarboxylase (4.1.1.28), i.e. tryptophane-decarboxylase
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/20—Animals treated with compounds which are neither proteins nor nucleic acids
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
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    • C12N2740/00—Reverse transcribing RNA viruses
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    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6072—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses
    • C12N2810/6081—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses rhabdoviridae, e.g. VSV

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  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
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  • Virology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2021555083A 2019-03-10 2020-03-10 パーキンソン病を処置するための遺伝子治療組成物および方法 Pending JP2022525136A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962816170P 2019-03-10 2019-03-10
US62/816,170 2019-03-10
US201962871007P 2019-07-05 2019-07-05
US62/871,007 2019-07-05
PCT/IB2020/052073 WO2020183374A1 (en) 2019-03-10 2020-03-10 Gene therapy compositions and methods for treating parkinson's disease

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JP2022525136A true JP2022525136A (ja) 2022-05-11
JP2022525136A5 JP2022525136A5 (enExample) 2023-03-06

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US (1) US20220175962A1 (enExample)
EP (1) EP3937985A1 (enExample)
JP (1) JP2022525136A (enExample)
KR (1) KR20220034719A (enExample)
CN (1) CN115835887A (enExample)
AU (1) AU2020236735A1 (enExample)
CA (1) CA3133188A1 (enExample)
WO (1) WO2020183374A1 (enExample)

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WO2024251679A1 (fr) * 2023-06-06 2024-12-12 Centre D'etude Des Cellules Souches (Cecs) Vecteur lentiviral pour la synthese de dopamine

Citations (1)

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JP2014532402A (ja) * 2011-10-28 2014-12-08 オックスフォード バイオメディカ （ユーケー） リミテッド 構築物

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US20220175962A1 (en) 2022-06-09
KR20220034719A (ko) 2022-03-18
CA3133188A1 (en) 2020-09-17
CN115835887A (zh) 2023-03-21
AU2020236735A1 (en) 2021-10-14
WO2020183374A1 (en) 2020-09-17
EP3937985A1 (en) 2022-01-19

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