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

    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (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 (6)

Country Link
KR (1) KR20240133725A (en)
CN (1) CN118679257A (en)
AU (1) AU2022417615A1 (en)
CA (1) CA3240593A1 (en)
IL (1) IL313726A (en)
WO (1) WO2023122800A1 (en)

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
WO1998010090A1 (en) 1996-09-06 1998-03-12 St. Elizabeth's Medical Center Of Boston, Inc. Gata-6 transcription factor: compositions and methods
EP2595664B1 (en) * 2010-07-19 2018-10-17 Ionis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
US20140212873A1 (en) * 2011-06-24 2014-07-31 La Trobe University Treatment and diagnosis of epigenetic disorders and conditions
RS64622B1 (en) 2012-05-25 2023-10-31 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
BR112015008708A2 (en) 2012-10-23 2017-09-26 Toolgen Inc a target DNA cleavage composition comprising a target DNA specific guide and nucleic acid encoding cas protein or cas protein and use thereof
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
DK2898075T3 (en) 2012-12-12 2016-06-27 Broad Inst Inc CONSTRUCTION AND OPTIMIZATION OF IMPROVED SYSTEMS, PROCEDURES AND ENZYME COMPOSITIONS FOR SEQUENCE MANIPULATION
PT2771468E (en) 2012-12-12 2015-06-02 Harvard College Engineering of systems, methods and optimized guide compositions for sequence manipulation
BR122021008308B1 (en) 2012-12-12 2022-12-27 The Broad Institute, Inc. CRISPR-CAS COMPONENT SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION
PL2931898T3 (en) 2012-12-12 2016-09-30 Le Cong Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2015023937A1 (en) * 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Heterochromatin forming non-coding rnas
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
CA3130854A1 (en) * 2019-02-26 2020-09-03 Nogra Pharma Limited Fragile x mental retardation protein interfering oligonucleotides and methods of using same
AU2020269398A1 (en) * 2019-05-07 2021-11-25 Ecole Polytechnique Federale De Lausanne (Epfl) 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
CN118679257A (en) 2024-09-20
WO2023122800A1 (en) 2023-06-29
CA3240593A1 (en) 2023-06-29
AU2022417615A1 (en) 2024-06-27
KR20240133725A (en) 2024-09-04

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