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

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US10370432B2 (en) 2014-10-03 2019-08-06 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
CA3044291A1 (en) 2016-11-17 2018-05-24 Nationwide Children's Hospital Inc. Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2
US12138298B2 (en) 2017-01-30 2024-11-12 Brainvectis Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
AU2019354995B2 (en) 2018-10-02 2024-12-12 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
US20220202960A1 (en) * 2019-04-24 2022-06-30 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
JP7624410B2 (ja) * 2019-05-21 2025-01-30 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル レット症候群の治療におけるコレステロール 24-ヒドロラーゼのための発現ベクター
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
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
WO2021183433A1 (en) * 2020-03-09 2021-09-16 University Of Massachusetts Gene replacement therapy for foxg1 syndrome
US20230203102A1 (en) * 2020-05-13 2023-06-29 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
EP4172330A1 (en) * 2020-06-30 2023-05-03 The University Court Of The University of Edinburgh Transgene expression system
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
CN112194706B (zh) * 2020-09-30 2022-03-08 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
KR20240113624A (ko) 2021-11-02 2024-07-22 보이저 테라퓨틱스, 인크. Aav 캡시드 변이체 및 이의 용도
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
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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IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
CA2915124C (en) 2001-11-13 2018-08-14 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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JP2011501760A (ja) 2007-10-23 2011-01-13 ノバルティス アーゲー 呼吸器疾患の処置のためのtrkb抗体の使用
CN102015769B (zh) 2008-01-17 2014-12-10 Irm责任有限公司 改进的抗-trkb抗体
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US10975391B2 (en) * 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
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Also Published As

Publication number Publication date
US20230416779A1 (en) 2023-12-28
BR112019025732A2 (pt) 2020-06-30
JP2020522269A (ja) 2020-07-30
IL271170A (en) 2020-01-30
EP3635122A1 (en) 2020-04-15
KR20200015701A (ko) 2020-02-12
SG11201911737PA (en) 2020-01-30
CN111065742A (zh) 2020-04-24
US11680275B2 (en) 2023-06-20
JP2023164447A (ja) 2023-11-10
EP3635122A4 (en) 2021-03-31
AU2018281145A1 (en) 2020-01-02
US20200181646A1 (en) 2020-06-11
WO2018226785A1 (en) 2018-12-13
CL2019003582A1 (es) 2020-09-25
MX2019014760A (es) 2020-08-03
EA201992882A1 (ru) 2020-05-25
CO2019014840A2 (es) 2020-01-17

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