EA201992882A1 - САМОРЕГУЛИРУЕМЫЕ ВЕКТОРЫ НА ОСНОВЕ AAV ДЛЯ БЕЗОПАСНОЙ ЭКСПРЕССИИ MeCP2 ПРИ СИНДРОМЕ PETTA - Google Patents

САМОРЕГУЛИРУЕМЫЕ ВЕКТОРЫ НА ОСНОВЕ AAV ДЛЯ БЕЗОПАСНОЙ ЭКСПРЕССИИ MeCP2 ПРИ СИНДРОМЕ PETTA

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Publication number
EA201992882A1
EA201992882A1 EA201992882A EA201992882A EA201992882A1 EA 201992882 A1 EA201992882 A1 EA 201992882A1 EA 201992882 A EA201992882 A EA 201992882A EA 201992882 A EA201992882 A EA 201992882A EA 201992882 A1 EA201992882 A1 EA 201992882A1
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Eurasian Patent Office
Prior art keywords
self
syndrome
petta
aav
safe
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EA201992882A
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English (en)
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Мигель Сена Эстевес
Гуанпин Гао
Майкл Р. Грин
Дэн Ван
Тесса Мерседес Симоне
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Юниверсити Оф Массачусеттс
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Publication of EA201992882A1 publication Critical patent/EA201992882A1/ru

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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
    • C12N15/63—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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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)

Abstract

В соответствии с некоторыми аспектами настоящее изобретение относится к композициям и способам конструирования трансгена. В соответствии с некоторыми вариантами осуществления изобретение относится к саморегулируемым рекомбинантным нуклеиновым кислотам, вирусным векторам и фармацевтическим композициям, содержащим трансген MeCP2. В соответствии с некоторыми вариантами осуществления композиции и способы, описанные в настоящем изобретении, являются пригодными для лечения заболеваний и нарушений, ассоциированных с мутацией с потерей функции, например синдрома Ретта.
EA201992882A 2017-06-06 2018-06-06 САМОРЕГУЛИРУЕМЫЕ ВЕКТОРЫ НА ОСНОВЕ AAV ДЛЯ БЕЗОПАСНОЙ ЭКСПРЕССИИ MeCP2 ПРИ СИНДРОМЕ PETTA EA201992882A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762516060P 2017-06-06 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

Publications (1)

Publication Number Publication Date
EA201992882A1 true EA201992882A1 (ru) 2020-05-25

Family

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Application Number Title Priority Date Filing Date
EA201992882A EA201992882A1 (ru) 2017-06-06 2018-06-06 САМОРЕГУЛИРУЕМЫЕ ВЕКТОРЫ НА ОСНОВЕ AAV ДЛЯ БЕЗОПАСНОЙ ЭКСПРЕССИИ MeCP2 ПРИ СИНДРОМЕ PETTA

Country Status (15)

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

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AU2017362491B2 (en) * 2016-11-17 2023-02-02 Nationwide Children's Hospital Inc. Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
MX2020010466A (es) 2018-04-03 2021-01-08 Vectores de virus que evitan anticuerpos.
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
KR20220011616A (ko) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 재조합 아데노 관련 바이러스 벡터
CN114026236A (zh) * 2019-04-24 2022-02-08 宾夕法尼亚州大学信托人 可用于治疗雷特综合征的组合物
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WO2021183433A1 (en) * 2020-03-09 2021-09-16 University Of Massachusetts Gene replacement therapy for foxg1 syndrome
BR112022023106A2 (pt) * 2020-05-13 2023-01-17 Voyager Therapeutics Inc Redirecionamento de tropismo de capsídeos de aav
KR20230068444A (ko) * 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터
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
GB202201242D0 (en) * 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
WO2024194491A1 (en) * 2023-03-22 2024-09-26 Ospedale San Raffaele S.R.L. Gene therapy

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JP2020522269A (ja) 2020-07-30
WO2018226785A1 (en) 2018-12-13
US11680275B2 (en) 2023-06-20
US20200181646A1 (en) 2020-06-11
CN111065742A (zh) 2020-04-24
MX2019014760A (es) 2020-08-03
EP3635122A1 (en) 2020-04-15
CA3066623A1 (en) 2019-12-13
CL2019003582A1 (es) 2020-09-25
EP3635122A4 (en) 2021-03-31
AU2018281145A1 (en) 2020-01-02
US20230416779A1 (en) 2023-12-28
IL271170A (en) 2020-01-30
BR112019025732A2 (pt) 2020-06-30
SG11201911737PA (en) 2020-01-30
KR20200015701A (ko) 2020-02-12
CO2019014840A2 (es) 2020-01-17
JP2023164447A (ja) 2023-11-10

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