CL2019003582A1 - Vectores aav autoreguladores para la correcta expresión de mecp2 en el sindrome de rett. - Google Patents

Vectores aav autoreguladores para la correcta expresión de mecp2 en el sindrome de rett.

Info

Publication number
CL2019003582A1
CL2019003582A1 CL2019003582A CL2019003582A CL2019003582A1 CL 2019003582 A1 CL2019003582 A1 CL 2019003582A1 CL 2019003582 A CL2019003582 A CL 2019003582A CL 2019003582 A CL2019003582 A CL 2019003582A CL 2019003582 A1 CL2019003582 A1 CL 2019003582A1
Authority
CL
Chile
Prior art keywords
mecp2
rett syndrome
autoregulatory
aav vectors
correct expression
Prior art date
Application number
CL2019003582A
Other languages
English (en)
Spanish (es)
Inventor
Miguel Sena Esteves
Guangping Gao
Michael R Green
Dan Wang
Tessa Mercedes Simone
Original Assignee
Univ Massachusetts
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Massachusetts filed Critical Univ Massachusetts
Publication of CL2019003582A1 publication Critical patent/CL2019003582A1/es

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Classifications

    • 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

Landscapes

  • 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)
CL2019003582A 2017-06-06 2019-12-06 Vectores aav autoreguladores para la correcta expresión de mecp2 en el sindrome de rett. CL2019003582A1 (es)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
CL2019003582A1 true CL2019003582A1 (es) 2020-09-25

Family

ID=64567243

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2019003582A CL2019003582A1 (es) 2017-06-06 2019-12-06 Vectores aav autoreguladores para la correcta expresión de mecp2 en el sindrome de rett.

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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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
CA3066623A1 (en) 2019-12-13
MX2019014760A (es) 2020-08-03
EA201992882A1 (ru) 2020-05-25
CO2019014840A2 (es) 2020-01-17

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