CN111065742A - 用于MECP2在Rett综合征中安全表达的自调节AAV载体 - Google Patents

用于MECP2在Rett综合征中安全表达的自调节AAV载体 Download PDF

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Publication number
CN111065742A
CN111065742A CN201880051148.4A CN201880051148A CN111065742A CN 111065742 A CN111065742 A CN 111065742A CN 201880051148 A CN201880051148 A CN 201880051148A CN 111065742 A CN111065742 A CN 111065742A
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mir
hsa
mirna
nucleic acid
expression
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M·S·埃斯特维斯
高光平
M·R·格林
王丹
T·M·西蒙尼
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University of Massachusetts Amherst
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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
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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 nucleic acids [NA]
    • 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
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    • 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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    • 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
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    • 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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  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (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)
CN201880051148.4A 2017-06-06 2018-06-06 用于MECP2在Rett综合征中安全表达的自调节AAV载体 Pending CN111065742A (zh)

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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CN111065742A true CN111065742A (zh) 2020-04-24

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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)

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CN113754727A (zh) * 2020-09-30 2021-12-07 广州派真生物技术有限公司 腺相关病毒突变体及其应用
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
WO2024251232A1 (zh) * 2023-06-09 2024-12-12 仁景(苏州)生物科技有限公司 表达可调控的工程化rna分子

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WO2018138371A1 (en) 2017-01-30 2018-08-02 Brainvectis Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
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KR20220003553A (ko) * 2019-04-24 2022-01-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 레트 증후군의 치료에 유용한 조성물
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
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療ｃ型尼曼—匹克病之腺相關病毒載體
JP7702949B2 (ja) * 2019-12-05 2025-07-04 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム ヒトｍｅｃｐ２遺伝子を発現するように設計されたトランスジーンカセット
WO2021183433A1 (en) 2020-03-09 2021-09-16 University Of Massachusetts Gene replacement therapy for foxg1 syndrome
CN116096734A (zh) * 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向
IL299414A (en) * 2020-06-30 2023-02-01 Univ Court Univ Of Edinburgh Transgene expression system
CN116745409A (zh) * 2020-08-19 2023-09-12 萨雷普塔治疗公司 用于治疗雷特综合征的腺相关病毒载体
WO2022094078A1 (en) * 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
US20250001012A1 (en) 2021-11-02 2025-01-02 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

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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113754727A (zh) * 2020-09-30 2021-12-07 广州派真生物技术有限公司 腺相关病毒突变体及其应用
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
WO2024251232A1 (zh) * 2023-06-09 2024-12-12 仁景(苏州)生物科技有限公司 表达可调控的工程化rna分子

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BR112019025732A2 (pt) 2020-06-30
CO2019014840A2 (es) 2020-01-17
IL271170A (en) 2020-01-30
EA201992882A1 (ru) 2020-05-25
US20200181646A1 (en) 2020-06-11
US11680275B2 (en) 2023-06-20
KR20200015701A (ko) 2020-02-12
EP3635122A1 (en) 2020-04-15
AU2018281145A1 (en) 2020-01-02
EP3635122A4 (en) 2021-03-31
JP2023164447A (ja) 2023-11-10
JP2020522269A (ja) 2020-07-30
WO2018226785A1 (en) 2018-12-13
MX2019014760A (es) 2020-08-03
CA3066623A1 (en) 2019-12-13
US20230416779A1 (en) 2023-12-28
SG11201911737PA (en) 2020-01-30
CL2019003582A1 (es) 2020-09-25

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