JP2020522269A - レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター - Google Patents

レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター Download PDF

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JP2020522269A
JP2020522269A JP2019567677A JP2019567677A JP2020522269A JP 2020522269 A JP2020522269 A JP 2020522269A JP 2019567677 A JP2019567677 A JP 2019567677A JP 2019567677 A JP2019567677 A JP 2019567677A JP 2020522269 A JP2020522269 A JP 2020522269A
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mir
hsa
mirna
nucleic acid
mecp2
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JP2020522269A5 (enExample
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エステベス，ミゲル，セナ
ガオ，グワンピン
グリーン，マイケル，アール．
ワン，ダン
シモーヌ，テサ，メルセデス
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ユニバーシティ オブ マサチューセッツ
ユニバーシティ オブ マサチューセッツ
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Publication of JP2020522269A publication Critical patent/JP2020522269A/ja
Publication of JP2020522269A5 publication Critical patent/JP2020522269A5/ja
Priority to JP2023135189A priority Critical patent/JP2023164447A/ja
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/41—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/10—Production naturally occurring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (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)
JP2019567677A 2017-06-06 2018-06-06 レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター Pending JP2020522269A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023135189A JP2023164447A (ja) 2017-06-06 2023-08-23 レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762516060P 2017-06-06 2017-06-06
US62/516,060 2017-06-06
PCT/US2018/036200 WO2018226785A1 (en) 2017-06-06 2018-06-06 Self-regulating aav vectors for safe expression of mecp2 in rett syndrome

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JP2023135189A Division JP2023164447A (ja) 2017-06-06 2023-08-23 レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター

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JP2020522269A true JP2020522269A (ja) 2020-07-30
JP2020522269A5 JP2020522269A5 (enExample) 2021-07-26

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JP2019567677A Pending JP2020522269A (ja) 2017-06-06 2018-06-06 レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター
JP2023135189A Pending JP2023164447A (ja) 2017-06-06 2023-08-23 レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター

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US (2) US11680275B2 (enExample)
EP (1) EP3635122A4 (enExample)
JP (2) JP2020522269A (enExample)
KR (1) KR20200015701A (enExample)
CN (1) CN111065742A (enExample)
AU (1) AU2018281145A1 (enExample)
BR (1) BR112019025732A2 (enExample)
CA (1) CA3066623A1 (enExample)
CL (1) CL2019003582A1 (enExample)
CO (1) CO2019014840A2 (enExample)
EA (1) EA201992882A1 (enExample)
IL (1) IL271170A (enExample)
MX (1) MX2019014760A (enExample)
SG (1) SG11201911737PA (enExample)
WO (1) WO2018226785A1 (enExample)

Cited By (1)

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JP2023542614A (ja) * 2020-08-19 2023-10-11 サレプタ セラピューティクス， インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター

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WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
US11583564B2 (en) 2016-11-17 2023-02-21 Nationwide Children's Hospital, Inc. Intrathecal delivery of recombinant adeno-associated virus encoding Methyl-CpG binding protein 2
ES2944358T3 (es) 2017-01-30 2023-06-20 Brainvectis Vector de expresión para la colesterol 24-hidrolasa en terapia de ataxias espinocerebelosas de repetición de poliglutamina
CN112272672A (zh) 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
CN119684417A (zh) 2018-10-02 2025-03-25 沃雅戈治疗公司 重新定向aav衣壳的嗜性
SG11202110146VA (en) 2019-03-21 2021-10-28 Stridebio Inc Recombinant adeno-associated virus vectors
AU2020261051A1 (en) * 2019-04-24 2021-11-04 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of Rett syndrome
EP3972631B1 (en) * 2019-05-21 2024-07-10 Institut National de la Santé et de la Recherche Médicale (INSERM) Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome
EP4045525A1 (en) 2019-10-17 2022-08-24 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
IL293431A (en) * 2019-12-05 2022-07-01 Univ Texas Transgene cassettes designed to express human mecp2 gene
EP4117735A4 (en) * 2020-03-09 2024-04-10 University of Massachusetts GENE REPLACEMENT THERAPY FOR FOXG1 SYNDROME
IL298001A (en) * 2020-05-13 2023-01-01 Voyager Therapeutics Inc Redirecting tropism of adeno-associated virus capsids
IL299414A (en) * 2020-06-30 2023-02-01 Univ Court Univ Of Edinburgh Transgene expression system
CN113717248B (zh) * 2020-09-30 2022-07-08 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2022094078A1 (en) * 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
WO2023081648A1 (en) 2021-11-02 2023-05-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
GB202201242D0 (en) * 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
AU2024241563A1 (en) * 2023-03-22 2025-10-09 Consiglio Nazionale Delle Ricerche Gene therapy
WO2024235164A1 (zh) * 2023-05-15 2024-11-21 上海金珂博生物技术有限公司 一种Rett综合症的基因治疗
TW202513796A (zh) * 2023-06-09 2025-04-01 大陸商仁景（蘇州）生物科技有限公司 表達可調控的工程化ｒｎａ分子及其用途

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US11680275B2 (en) 2023-06-20
AU2018281145A1 (en) 2020-01-02
US20230416779A1 (en) 2023-12-28
SG11201911737PA (en) 2020-01-30
US20200181646A1 (en) 2020-06-11
CN111065742A (zh) 2020-04-24
WO2018226785A1 (en) 2018-12-13
EP3635122A1 (en) 2020-04-15
MX2019014760A (es) 2020-08-03
EP3635122A4 (en) 2021-03-31
IL271170A (en) 2020-01-30
CA3066623A1 (en) 2019-12-13
BR112019025732A2 (pt) 2020-06-30
KR20200015701A (ko) 2020-02-12
CL2019003582A1 (es) 2020-09-25
CO2019014840A2 (es) 2020-01-17
EA201992882A1 (ru) 2020-05-25
JP2023164447A (ja) 2023-11-10

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