MX2019014760A - Vectores de aav autoregulables para la expresión segura de mecp2 en el síndrome de rett. - Google Patents

Vectores de aav autoregulables para la expresión segura de mecp2 en el síndrome de rett.

Info

Publication number
MX2019014760A
MX2019014760A MX2019014760A MX2019014760A MX2019014760A MX 2019014760 A MX2019014760 A MX 2019014760A MX 2019014760 A MX2019014760 A MX 2019014760A MX 2019014760 A MX2019014760 A MX 2019014760A MX 2019014760 A MX2019014760 A MX 2019014760A
Authority
MX
Mexico
Prior art keywords
mecp2
rett syndrome
self
aav vectors
compositions
Prior art date
Application number
MX2019014760A
Other languages
English (en)
Spanish (es)
Inventor
Miguel Sena Esteves
Guangping Gao
Dan Wang
Tessa Mercedes Simone
Michael R Green
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 MX2019014760A publication Critical patent/MX2019014760A/es

Links

Classifications

    • 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
    • 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
    • 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)
MX2019014760A 2017-06-06 2018-06-06 Vectores de aav autoregulables para la expresión segura de mecp2 en el síndrome de rett. MX2019014760A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762516060P 2017-06-06 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
MX2019014760A true MX2019014760A (es) 2020-08-03

Family

ID=64567243

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019014760A MX2019014760A (es) 2017-06-06 2018-06-06 Vectores de aav autoregulables para la expresión segura de mecp2 en el síndrome 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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WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
EP3541429A4 (en) 2016-11-17 2020-08-12 Nationwide Children's Hospital, Inc. INTRATHECAL ADMINISTRATION OF RECOMBINANT ADENO-ASSOCIATED VIRUS CODING FOR METHYL-CPG-BINDING PROTEIN 2
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
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ，インコーポレイテッド 抗体を回避するウイルスベクター
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
EP3861010A1 (en) 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
AU2020241888A1 (en) 2019-03-21 2021-09-30 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors
JP2022530095A (ja) * 2019-04-24 2022-06-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア レット症候群の治療に有用な組成物
WO2020234363A2 (en) * 2019-05-21 2020-11-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
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
PE20230767A1 (es) * 2020-05-13 2023-05-09 Voyager Therapeutics Inc Redireccion del tropismo de las capsides de aav
KR20230029891A (ko) * 2020-06-30 2023-03-03 더 유니버시티 코트 오브 더 유니버시티 오브 에딘버그 전이유전자 발현 시스템
AR123288A1 (es) 2020-08-19 2022-11-16 Stridebio Inc Vectores de virus adenoasociados para el tratamiento del síndrome de rett
CN113754728B (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
MX2024004936A (es) 2021-11-02 2024-05-08 Voyager Therapeutics Inc Variantes de capsides de aav y sus usos.
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
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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US20040106566A1 (en) * 2002-05-17 2004-06-03 Shi-Lung Lin RNA-splicing and processing-directed gene silencing and the relative applications thereof
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Also Published As

Publication number Publication date
CA3066623A1 (en) 2019-12-13
CO2019014840A2 (es) 2020-01-17
US11680275B2 (en) 2023-06-20
US20200181646A1 (en) 2020-06-11
BR112019025732A2 (pt) 2020-06-30
WO2018226785A1 (en) 2018-12-13
EA201992882A1 (ru) 2020-05-25
CN111065742A (zh) 2020-04-24
US20230416779A1 (en) 2023-12-28
KR20200015701A (ko) 2020-02-12
CL2019003582A1 (es) 2020-09-25
IL271170A (en) 2020-01-30
AU2018281145A1 (en) 2020-01-02
SG11201911737PA (en) 2020-01-30
JP2023164447A (ja) 2023-11-10
EP3635122A1 (en) 2020-04-15
JP2020522269A (ja) 2020-07-30
EP3635122A4 (en) 2021-03-31

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