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IL313726A - Therapeutic treatment for fragile x-associated disorder - Google Patents

Therapeutic treatment for fragile x-associated disorder

Info

Publication number
IL313726A
IL313726A IL313726A IL31372624A IL313726A IL 313726 A IL313726 A IL 313726A IL 313726 A IL313726 A IL 313726A IL 31372624 A IL31372624 A IL 31372624A IL 313726 A IL313726 A IL 313726A
Authority
IL
Israel
Prior art keywords
fragile
therapeutic treatment
associated disorder
disorder
therapeutic
Prior art date
Application number
IL313726A
Other languages
Hebrew (he)
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 IL313726A publication Critical patent/IL313726A/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • 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
    • C12N2310/11Antisense
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
IL313726A 2021-12-23 2022-12-23 Therapeutic treatment for fragile x-associated disorder IL313726A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163265989P 2021-12-23 2021-12-23
PCT/US2022/082380 WO2023122800A1 (en) 2021-12-23 2022-12-23 Therapeutic treatment for fragile x-associated disorder

Publications (1)

Publication Number Publication Date
IL313726A true IL313726A (en) 2024-08-01

Family

ID=85199427

Family Applications (1)

Application Number Title Priority Date Filing Date
IL313726A IL313726A (en) 2021-12-23 2022-12-23 Therapeutic treatment for fragile x-associated disorder

Country Status (9)

Country Link
EP (1) EP4453212A1 (en)
JP (1) JP2025501755A (en)
KR (1) KR20240133725A (en)
CN (1) CN118679257A (en)
AU (1) AU2022417615A1 (en)
CA (1) CA3240593A1 (en)
IL (1) IL313726A (en)
MX (1) MX2024007908A (en)
WO (1) WO2023122800A1 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025240947A1 (en) * 2024-05-17 2025-11-20 University Of Massachusetts Therapeutic treatment for fragile x-associated disorder

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3854480A (en) 1969-04-01 1974-12-17 Alza Corp Drug-delivery system
US4452775A (en) 1982-12-03 1984-06-05 Syntex (U.S.A.) Inc. Cholesterol matrix delivery system for sustained release of macromolecules
US5039660A (en) 1988-03-02 1991-08-13 Endocon, Inc. Partially fused peptide pellet
CA2264485A1 (en) 1996-09-06 1998-03-12 St. Elizabeth's Medical Center Of Boston, Inc. Gata-6 transcription factor: compositions and methods
EP3031920B1 (en) * 2010-07-19 2019-08-21 Ionis Pharmaceuticals, Inc. Modulation of dystrophia myotonica-protein kinase (dmpk) expression
EP2723902A4 (en) * 2011-06-24 2015-02-18 Murdoch Childrens Res Inst TREATMENT AND DIAGNOSIS OF EPIGENETIC DISORDERS AND CONDITIONS
PT3241902T (en) 2012-05-25 2018-05-28 Univ California METHODS AND COMPOSITIONS FOR MODIFICATION OF TARGETED TARGET DNA BY RNA AND FOR MODULATION DIRECTED BY TRANSCRIPTION RNA
WO2014065596A1 (en) 2012-10-23 2014-05-01 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
KR20150105634A (en) 2012-12-12 2015-09-17 더 브로드 인스티튜트, 인코퍼레이티드 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
ES2553782T3 (en) 2012-12-12 2015-12-11 The Broad Institute, Inc. Systems engineering, methods and guide compositions optimized for sequence manipulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
JP2016528258A (en) * 2013-08-16 2016-09-15 ラナ セラピューティクス インコーポレイテッド Heterochromatin forming non-coding RNA
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2018170290A1 (en) * 2017-03-15 2018-09-20 Fulcrum Therapeutics, Inc. Compositions and methods for increasing fmr1 expression
GB2574243A (en) * 2018-05-31 2019-12-04 The Govening Council Of The Univ Of Toronto Adeno-associated viral vector-mediated gene therapy for treating fragile X-associated disorders
AU2020228134A1 (en) * 2019-02-26 2021-10-14 Nogra Pharma Limited Fragile X mental retardation protein interfering oligonucleotides and methods of using same
CN120022385A (en) * 2019-05-07 2025-05-23 洛桑联邦理工学院 FMRP and cancer treatment
CN113151406B (en) * 2021-03-25 2022-06-21 深圳会众生物技术有限公司 FMR1 gene CGG repetition number and methylation detection kit and detection method

Also Published As

Publication number Publication date
MX2024007908A (en) 2024-09-10
AU2022417615A1 (en) 2024-06-27
JP2025501755A (en) 2025-01-23
CN118679257A (en) 2024-09-20
WO2023122800A1 (en) 2023-06-29
KR20240133725A (en) 2024-09-04
EP4453212A1 (en) 2024-10-30
CA3240593A1 (en) 2023-06-29

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