CA3066623A1 - Self-regulating aav vectors for safe expression of mecp2 in rett syndrome - Google Patents

Self-regulating aav vectors for safe expression of mecp2 in rett syndrome Download PDF

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Publication number
CA3066623A1
CA3066623A1 CA3066623A CA3066623A CA3066623A1 CA 3066623 A1 CA3066623 A1 CA 3066623A1 CA 3066623 A CA3066623 A CA 3066623A CA 3066623 A CA3066623 A CA 3066623A CA 3066623 A1 CA3066623 A1 CA 3066623A1
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Prior art keywords
mir
hsa
mirna
nucleic acid
mecp2
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Pending
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CA3066623A
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English (en)
French (fr)
Inventor
Miguel Sena ESTEVES
Guangping Gao
Michael R. Green
Dan Wang
Tessa Mercedes Simone
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University of Massachusetts Amherst
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University of Massachusetts Amherst
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Application filed by University of Massachusetts Amherst filed Critical University of Massachusetts Amherst
Publication of CA3066623A1 publication Critical patent/CA3066623A1/en
Pending legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/40Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/41Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/10Production naturally occurring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/007Vectors comprising a special translation-regulating system cell or tissue specific
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/10Vectors 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)
CA3066623A 2017-06-06 2018-06-06 Self-regulating aav vectors for safe expression of mecp2 in rett syndrome Pending CA3066623A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3066623A1 true CA3066623A1 (en) 2019-12-13

Family

ID=64567243

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3066623A Pending CA3066623A1 (en) 2017-06-06 2018-06-06 Self-regulating aav vectors for safe expression of mecp2 in rett syndrome

Country Status (15)

Country Link
US (2) US11680275B2 (cg-RX-API-DMAC7.html)
EP (1) EP3635122A4 (cg-RX-API-DMAC7.html)
JP (2) JP2020522269A (cg-RX-API-DMAC7.html)
KR (1) KR20200015701A (cg-RX-API-DMAC7.html)
CN (1) CN111065742A (cg-RX-API-DMAC7.html)
AU (1) AU2018281145A1 (cg-RX-API-DMAC7.html)
BR (1) BR112019025732A2 (cg-RX-API-DMAC7.html)
CA (1) CA3066623A1 (cg-RX-API-DMAC7.html)
CL (1) CL2019003582A1 (cg-RX-API-DMAC7.html)
CO (1) CO2019014840A2 (cg-RX-API-DMAC7.html)
EA (1) EA201992882A1 (cg-RX-API-DMAC7.html)
IL (1) IL271170A (cg-RX-API-DMAC7.html)
MX (1) MX2019014760A (cg-RX-API-DMAC7.html)
SG (1) SG11201911737PA (cg-RX-API-DMAC7.html)
WO (1) WO2018226785A1 (cg-RX-API-DMAC7.html)

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WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
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CN110325219B (zh) * 2016-11-17 2024-03-26 全国儿童医院公司 编码甲基-CpG结合蛋白2的重组腺相关病毒的鞘内递送
EP3574091B1 (en) 2017-01-30 2023-02-22 Brainvectis Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
EA202092362A1 (ru) 2018-04-03 2021-01-11 Страйдбайо, Инк. Вирусные векторы для нацеливания на ткани глаза
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
AU2019354995B2 (en) 2018-10-02 2024-12-12 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
US20220202960A1 (en) * 2019-04-24 2022-06-30 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
CN114555813A (zh) * 2019-05-21 2022-05-27 国家医疗保健研究所 治疗雷特综合征的胆固醇24-水解酶的表达载体
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
WO2021113634A1 (en) * 2019-12-05 2021-06-10 The Board Of Regents Of The University Of Texas Transgene cassettes designed to express a human mecp2 gene
US12497612B2 (en) 2020-03-09 2025-12-16 University Of Massachusetts Gene replacement therapy for FOXG1 syndrome
PE20230767A1 (es) * 2020-05-13 2023-05-09 Voyager Therapeutics Inc Redireccion del tropismo de las capsides de aav
AU2021300615A1 (en) * 2020-06-30 2023-02-02 The University Court Of The University Of Edinburgh Transgene expression system
US12104163B2 (en) * 2020-08-19 2024-10-01 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
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
IL311861A (en) 2021-11-02 2024-06-01 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
CN121219020A (zh) * 2023-03-22 2025-12-26 圣拉斐尔医院有限责任公司 基因疗法
CN121175427A (zh) * 2023-05-15 2025-12-19 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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Publication number Publication date
BR112019025732A2 (pt) 2020-06-30
WO2018226785A1 (en) 2018-12-13
CL2019003582A1 (es) 2020-09-25
JP2020522269A (ja) 2020-07-30
KR20200015701A (ko) 2020-02-12
MX2019014760A (es) 2020-08-03
EP3635122A1 (en) 2020-04-15
CO2019014840A2 (es) 2020-01-17
IL271170A (en) 2020-01-30
US11680275B2 (en) 2023-06-20
EP3635122A4 (en) 2021-03-31
AU2018281145A1 (en) 2020-01-02
JP2023164447A (ja) 2023-11-10
US20200181646A1 (en) 2020-06-11
US20230416779A1 (en) 2023-12-28
CN111065742A (zh) 2020-04-24
SG11201911737PA (en) 2020-01-30
EA201992882A1 (ru) 2020-05-25

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