CN114144203A - 源于显性变异基因的疾患的治疗剂 - Google Patents

源于显性变异基因的疾患的治疗剂 Download PDF

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CN114144203A
CN114144203A CN202080050265.6A CN202080050265A CN114144203A CN 114144203 A CN114144203 A CN 114144203A CN 202080050265 A CN202080050265 A CN 202080050265A CN 114144203 A CN114144203 A CN 114144203A
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gene
sequence
normal
therapeutic agent
rhodopsin
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高桥政代
大西晓士
恒川雄二
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RIKEN
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RIKEN
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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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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
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    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • 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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN202080050265.6A 2019-07-12 2020-07-10 源于显性变异基因的疾患的治疗剂 Pending CN114144203A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2019130199 2019-07-12
JP2019-130199 2019-07-12
PCT/JP2020/026956 WO2021010303A1 (ja) 2019-07-12 2020-07-10 顕性型変異遺伝子に由来する疾患の治療剤

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CN114144203A true CN114144203A (zh) 2022-03-04

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CN202080050265.6A Pending CN114144203A (zh) 2019-07-12 2020-07-10 源于显性变异基因的疾患的治疗剂

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US (1) US20220249698A1 (https=)
EP (1) EP3998086A4 (https=)
JP (1) JP7609436B2 (https=)
KR (1) KR20220034833A (https=)
CN (1) CN114144203A (https=)
AU (1) AU2020313004A1 (https=)
CA (1) CA3146497A1 (https=)
WO (1) WO2021010303A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023120536A1 (ja) * 2021-12-21 2023-06-29 国立大学法人東海国立大学機構 単一のａａｖベクターによるゲノム編集を用いた遺伝子治療
CN121263520A (zh) 2023-06-16 2026-01-02 株式会社维赛基因科技 锌指核酸酶、载体和应用它们的基因组编辑方法和视细胞制备方法

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
ES2788426T3 (es) * 2014-06-16 2020-10-21 Univ Johns Hopkins Composiciones y métodos para la expresión de ARNs de guía de CRISPR utilizando el promotor de H1
KR20190039703A (ko) * 2016-07-05 2019-04-15 더 존스 홉킨스 유니버시티 Crispr/cas9-기반 조성물 및 망막 변성을 치료하기 위한 방법
EP4321623A3 (en) 2016-07-15 2024-05-15 Salk Institute for Biological Studies Methods and compositions for genome editing in non-dividing cells
JP2022505139A (ja) * 2018-10-15 2022-01-14 フォンダッツィオーネ・テレソン ゲノム編集の方法及び構築物

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EP3998086A4 (en) 2023-08-30
CA3146497A1 (en) 2021-01-21
JP7609436B2 (ja) 2025-01-07
JPWO2021010303A1 (https=) 2021-01-21
KR20220034833A (ko) 2022-03-18
US20220249698A1 (en) 2022-08-11
WO2021010303A1 (ja) 2021-01-21
EP3998086A1 (en) 2022-05-18
AU2020313004A1 (en) 2022-03-10

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