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)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • 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)
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 (https=)
EP (1) EP3635122A4 (https=)
JP (2) JP2020522269A (https=)
KR (1) KR20200015701A (https=)
CN (1) CN111065742A (https=)
AU (1) AU2018281145A1 (https=)
BR (1) BR112019025732A2 (https=)
CA (1) CA3066623A1 (https=)
CL (1) CL2019003582A1 (https=)
CO (1) CO2019014840A2 (https=)
EA (1) EA201992882A1 (https=)
IL (1) IL271170A (https=)
MX (1) MX2019014760A (https=)
SG (1) SG11201911737PA (https=)
WO (1) WO2018226785A1 (https=)

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JP7106534B2 (ja) * 2016-11-17 2022-07-26 ネイションワイド チルドレンズ ホスピタル， インコーポレイテッド メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達
JP7618386B2 (ja) 2017-01-30 2025-01-21 ブレインヴェクティス ポリグルタミン反復脊髄小脳失調症の処置におけるコレステロール２４－ヒドロキシラーゼ（ｈｙｄｒｏｌａｓｅ）のための発現ベクター
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AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
CA3133889A1 (en) * 2019-04-24 2020-10-29 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
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EP4065600A1 (en) 2019-11-25 2022-10-05 Cornell University Apoe gene therapy
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
US12497612B2 (en) 2020-03-09 2025-12-16 University Of Massachusetts Gene replacement therapy for FOXG1 syndrome
MX2022014258A (es) * 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones para la reducción específica de un fármaco de la expresión de transgén.
TWI900553B (zh) 2020-05-13 2025-10-11 美商航海家醫療公司 Aav蛋白殼之趨性重定向
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AU2021328475A1 (en) * 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
CN113754727B (zh) * 2020-09-30 2022-07-12 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
EP4426716A1 (en) 2021-11-02 2024-09-11 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
EP4715058A1 (en) * 2023-05-15 2026-03-25 Genecombio Ltd. Gene therapy for rett syndrome
CN119101681A (zh) * 2023-06-09 2024-12-10 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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Also Published As

Publication number Publication date
JP2023164447A (ja) 2023-11-10
EP3635122A4 (en) 2021-03-31
CO2019014840A2 (es) 2020-01-17
MX2019014760A (es) 2020-08-03
KR20200015701A (ko) 2020-02-12
CN111065742A (zh) 2020-04-24
EA201992882A1 (ru) 2020-05-25
US20200181646A1 (en) 2020-06-11
JP2020522269A (ja) 2020-07-30
BR112019025732A2 (pt) 2020-06-30
US11680275B2 (en) 2023-06-20
EP3635122A1 (en) 2020-04-15
AU2018281145A1 (en) 2020-01-02
SG11201911737PA (en) 2020-01-30
WO2018226785A1 (en) 2018-12-13
IL271170A (en) 2020-01-30
CA3066623A1 (en) 2019-12-13
US20230416779A1 (en) 2023-12-28

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