JP2023545731A - 肢帯型２ｉ（ｌｇｍｄ２ｉ）を含むジストログリカノパチー障害を処置するためのフクチン関連タンパク質（ｆｋｒｐ）の治療的アデノ随伴ウイルス送達 - Google Patents

肢帯型２ｉ（ｌｇｍｄ２ｉ）を含むジストログリカノパチー障害を処置するためのフクチン関連タンパク質（ｆｋｒｐ）の治療的アデノ随伴ウイルス送達 Download PDF

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JP2023545731A
JP2023545731A JP2023521337A JP2023521337A JP2023545731A JP 2023545731 A JP2023545731 A JP 2023545731A JP 2023521337 A JP2023521337 A JP 2023521337A JP 2023521337 A JP2023521337 A JP 2023521337A JP 2023545731 A JP2023545731 A JP 2023545731A
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nucleic acid
promoter
seq
muscle
sequence
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JP2023545731A5 (https=
JPWO2022076556A5 (https=
Inventor
マーティン ケー． チルダーズ，
ジョージ オマル ヤネス－クーナ，
ゴンザレス， フアン マヌエル イグレシアス
シンクレア クーパー，
アントニア エブリピオティ，
マイケル エル． ロバーツ，
アンナ トレチャコヴァ，
レスター スアレス，
アン グエン，
シュウフイ ロウ，
シャオ シャオ，
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University of North Carolina at Chapel Hill
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University of North Carolina at Chapel Hill
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Publication of JP2023545731A5 publication Critical patent/JP2023545731A5/ja
Publication of JPWO2022076556A5 publication Critical patent/JPWO2022076556A5/ja
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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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
    • A01K2227/00—Animals characterised by species
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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
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    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • A61K48/005—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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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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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  • Wood Science & Technology (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2023521337A 2020-10-07 2021-10-06 肢帯型２ｉ（ｌｇｍｄ２ｉ）を含むジストログリカノパチー障害を処置するためのフクチン関連タンパク質（ｆｋｒｐ）の治療的アデノ随伴ウイルス送達 Pending JP2023545731A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063088757P 2020-10-07 2020-10-07
US63/088,757 2020-10-07
US202163214123P 2021-06-23 2021-06-23
US63/214,123 2021-06-23
US202163229726P 2021-08-05 2021-08-05
US63/229,726 2021-08-05
PCT/US2021/053768 WO2022076556A2 (en) 2020-10-07 2021-10-06 Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i)

Publications (3)

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JP2023545731A true JP2023545731A (ja) 2023-10-31
JP2023545731A5 JP2023545731A5 (https=) 2024-10-15
JPWO2022076556A5 JPWO2022076556A5 (https=) 2024-10-15

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US (1) US20230374542A1 (https=)
EP (1) EP4225382A4 (https=)
JP (1) JP2023545731A (https=)
AU (1) AU2021358957A1 (https=)
CA (1) CA3195177A1 (https=)
IL (1) IL301955A (https=)
WO (1) WO2022076556A2 (https=)

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JP2024524270A (ja) * 2021-06-23 2024-07-05 アスクバイオ・ユーケー・リミテッド 調節性核酸配列
US20250312398A1 (en) * 2022-04-29 2025-10-09 University Of Washington Method for expressing a muscle-specific gene and cassettes for same
GB202215198D0 (en) * 2022-10-14 2022-11-30 Univ Sheffield Gene therapy treatment
EP4665749A1 (en) * 2023-02-13 2025-12-24 Astrazeneca Ireland Limited Nucleic acids encoding bcl2-associated athanogene 3 (bag3) for gene therapy

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