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CA3266991A1 - Traitement du syndrome de rett - Google Patents

Traitement du syndrome de rett

Info

Publication number
CA3266991A1
CA3266991A1 CA3266991A CA3266991A CA3266991A1 CA 3266991 A1 CA3266991 A1 CA 3266991A1 CA 3266991 A CA3266991 A CA 3266991A CA 3266991 A CA3266991 A CA 3266991A CA 3266991 A1 CA3266991 A1 CA 3266991A1
Authority
CA
Canada
Prior art keywords
rett syndrome
syndrome therapy
therapy
rett
syndrome
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3266991A
Other languages
English (en)
Inventor
Benjamin Kleinstiver
Jacqueline Guy
Adrian Bird
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Edinburgh
General Hospital Corp
Original Assignee
University of Edinburgh
General Hospital Corp
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 University of Edinburgh, General Hospital Corp filed Critical University of Edinburgh
Publication of CA3266991A1 publication Critical patent/CA3266991A1/fr
Pending legal-status Critical Current

Links

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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3266991A 2022-09-08 2023-09-07 Traitement du syndrome de rett Pending CA3266991A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263375008P 2022-09-08 2022-09-08
PCT/GB2023/052315 WO2024052681A1 (fr) 2022-09-08 2023-09-07 Traitement du syndrome de rett

Publications (1)

Publication Number Publication Date
CA3266991A1 true CA3266991A1 (fr) 2024-03-14

Family

ID=88295935

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3266991A Pending CA3266991A1 (fr) 2022-09-08 2023-09-07 Traitement du syndrome de rett

Country Status (6)

Country Link
EP (1) EP4584376A1 (fr)
JP (1) JP2025530183A (fr)
KR (1) KR20250067157A (fr)
CN (1) CN120225674A (fr)
CA (1) CA3266991A1 (fr)
WO (1) WO2024052681A1 (fr)

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3177718B1 (fr) 2014-07-30 2022-03-16 President and Fellows of Harvard College Protéines cas9 comprenant des intéines dépendant de ligands
WO2017070633A2 (fr) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Protéines cas9 évoluées pour l'édition génétique
WO2018027078A1 (fr) 2016-08-03 2018-02-08 President And Fellows Of Harard College Éditeurs de nucléobases d'adénosine et utilisations associées
KR102687373B1 (ko) 2017-03-23 2024-07-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
EP3658573A1 (fr) 2017-07-28 2020-06-03 President and Fellows of Harvard College Procédés et compositions pour l'évolution d'éditeurs de bases à l'aide d'une évolution continue assistée par phage (pace)
EP3697906A1 (fr) 2017-10-16 2020-08-26 The Broad Institute, Inc. Utilisations d'éditeurs de bases adénosine
AU2019266326A1 (en) * 2018-05-11 2020-11-26 Beam Therapeutics Inc. Methods of editing single nucleotide polymorphism using programmable base editor systems
US11117812B2 (en) 2018-05-24 2021-09-14 Aqua-Aerobic Systems, Inc. System and method of solids conditioning in a filtration system
WO2020051360A1 (fr) 2018-09-05 2020-03-12 The Broad Institute, Inc. Édition de base pour le traitement du syndrome de hutchinson-gilford, progeria
US20220380740A1 (en) 2018-10-24 2022-12-01 The Broad Institute, Inc. Constructs for improved hdr-dependent genomic editing
US20220282275A1 (en) 2018-11-15 2022-09-08 The Broad Institute, Inc. G-to-t base editors and uses thereof
US20230140953A1 (en) * 2019-02-13 2023-05-11 Beam Therapeutics Inc. Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
EP3956349A1 (fr) 2019-04-17 2022-02-23 The Broad Institute, Inc. Éditeurs de base d'adénine présentant des effets hors cible réduits
CN120346348A (zh) * 2019-05-21 2025-07-22 比姆医疗股份有限公司 使用可编程碱基编辑器系统编辑单核苷酸多态性的方法

Also Published As

Publication number Publication date
JP2025530183A (ja) 2025-09-11
EP4584376A1 (fr) 2025-07-16
KR20250067157A (ko) 2025-05-14
WO2024052681A1 (fr) 2024-03-14
CN120225674A (zh) 2025-06-27

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