IL326502A - Usher syndrome-related gene modification - Google Patents

Usher syndrome-related gene modification

Info

Publication number
IL326502A
IL326502A IL326502A IL32650226A IL326502A IL 326502 A IL326502 A IL 326502A IL 326502 A IL326502 A IL 326502A IL 32650226 A IL32650226 A IL 32650226A IL 326502 A IL326502 A IL 326502A
Authority
IL
Israel
Prior art keywords
related gene
gene modification
usher syndrome
usher
syndrome
Prior art date
Application number
IL326502A
Other languages
Hebrew (he)
Original Assignee
Amber Bio Inc
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 Amber Bio Inc filed Critical Amber Bio Inc
Publication of IL326502A publication Critical patent/IL326502A/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
IL326502A 2023-08-15 2024-08-15 Usher syndrome-related gene modification IL326502A (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202363519830P 2023-08-15 2023-08-15
US202363602531P 2023-11-24 2023-11-24
US202463554908P 2024-02-16 2024-02-16
PCT/US2024/042505 WO2025038853A1 (en) 2023-08-15 2024-08-15 Usher syndrome-related gene modification

Publications (1)

Publication Number Publication Date
IL326502A true IL326502A (en) 2026-04-01

Family

ID=94633134

Family Applications (1)

Application Number Title Priority Date Filing Date
IL326502A IL326502A (en) 2023-08-15 2024-08-15 Usher syndrome-related gene modification

Country Status (3)

Country Link
AU (1) AU2024325107A1 (en)
IL (1) IL326502A (en)
WO (1) WO2025038853A1 (en)

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10139492B4 (en) * 2001-08-13 2004-06-09 Eul, Joachim, Dr. Process for the repair of a mutated RNA from a gene-defective DNA and for the targeted killing of tumor cells by RNA trans-splicing as well as process for the detection of naturally trans-spliced cellular RNA
US10131910B2 (en) * 2014-07-10 2018-11-20 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of Usher syndrome type 2
GB201616202D0 (en) * 2016-09-23 2016-11-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of eye deisease
EP3958872A4 (en) * 2019-04-25 2024-07-03 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE

Also Published As

Publication number Publication date
WO2025038853A1 (en) 2025-02-20
AU2024325107A1 (en) 2026-03-12

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