JP7418957B2 - タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 - Google Patents

タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 Download PDF

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JP7418957B2
JP7418957B2 JP2018525652A JP2018525652A JP7418957B2 JP 7418957 B2 JP7418957 B2 JP 7418957B2 JP 2018525652 A JP2018525652 A JP 2018525652A JP 2018525652 A JP2018525652 A JP 2018525652A JP 7418957 B2 JP7418957 B2 JP 7418957B2
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nucleic acid
titin
sequence
grna
gene
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ルイーズ ロディーノ－カラパック，
レイチェル ポッター，
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リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/39—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A—HUMAN NECESSITIES
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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
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JP2018525652A 2015-11-16 2016-11-15 タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 Active JP7418957B2 (ja)

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JP2021177441A JP2022009734A (ja) 2015-11-16 2021-10-29 タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法
JP2023190737A JP2023184679A (ja) 2015-11-16 2023-11-08 タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法

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US201562255887P 2015-11-16 2015-11-16
US62/255,887 2015-11-16
PCT/US2016/062052 WO2017087395A1 (en) 2015-11-16 2016-11-15 Materials and methods for treatment of titin-based myopathies and other titinopaties

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JP2023190737A Division JP2023184679A (ja) 2015-11-16 2023-11-08 タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法

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JP2018533959A JP2018533959A (ja) 2018-11-22
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JP2021177441A Pending JP2022009734A (ja) 2015-11-16 2021-10-29 タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法
JP2023190737A Withdrawn JP2023184679A (ja) 2015-11-16 2023-11-08 タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法

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JP2023190737A Withdrawn JP2023184679A (ja) 2015-11-16 2023-11-08 タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法

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US (2) US11617783B2 (enExample)
EP (1) EP3377042A4 (enExample)
JP (3) JP7418957B2 (enExample)
KR (1) KR102877920B1 (enExample)
CN (1) CN108463211B (enExample)
AU (1) AU2016358172B2 (enExample)
CA (1) CA3005633C (enExample)
CO (1) CO2018006133A2 (enExample)
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HK (1) HK1257955A1 (enExample)
IL (1) IL259441B2 (enExample)
MX (1) MX2018006116A (enExample)
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CN112543810A (zh) * 2018-06-29 2021-03-23 全国儿童医院研究所 治疗肢带型肌营养不良2a型的重组腺相关病毒产品和方法
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