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WO2024220980A3 - Dscam inhibitory nucleic acids and methods of use thereof - Google Patents

Dscam inhibitory nucleic acids and methods of use thereof Download PDF

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Publication number
WO2024220980A3
WO2024220980A3 PCT/US2024/025688 US2024025688W WO2024220980A3 WO 2024220980 A3 WO2024220980 A3 WO 2024220980A3 US 2024025688 W US2024025688 W US 2024025688W WO 2024220980 A3 WO2024220980 A3 WO 2024220980A3
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WO
WIPO (PCT)
Prior art keywords
methods
inhibitory nucleic
dscam
nucleic acids
subject
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
PCT/US2024/025688
Other languages
French (fr)
Other versions
WO2024220980A2 (en
Inventor
Jonathan R. Soucy
Petr BARANOV
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.)
Schepens Eye Research Institute Inc
Original Assignee
Schepens Eye Research Institute 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 Schepens Eye Research Institute Inc filed Critical Schepens Eye Research Institute Inc
Publication of WO2024220980A2 publication Critical patent/WO2024220980A2/en
Publication of WO2024220980A3 publication Critical patent/WO2024220980A3/en
Anticipated expiration legal-status Critical
Pending legal-status Critical Current

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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins

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  • 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)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

The disclosure features compositions and methods for the treatment of a subject. Disclosed herein are inhibitory nucleic acid molecules that suppress the expression of Down Syndrome Cell Adhesion Molecule (DSCAM), as well as mammalian cells and compositions comprising such inhibitory nucleic acid molecules. Also provided are methods for treating a subject (e.g., a subject having degeneration of the optic nerve (e.g., glaucoma)) with the compositions disclosed herein.
PCT/US2024/025688 2023-04-20 2024-04-22 Dscam inhibitory nucleic acids and methods of use thereof Pending WO2024220980A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363460855P 2023-04-20 2023-04-20
US63/460,855 2023-04-20

Publications (2)

Publication Number Publication Date
WO2024220980A2 WO2024220980A2 (en) 2024-10-24
WO2024220980A3 true WO2024220980A3 (en) 2024-12-05

Family

ID=93153423

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/025688 Pending WO2024220980A2 (en) 2023-04-20 2024-04-22 Dscam inhibitory nucleic acids and methods of use thereof

Country Status (1)

Country Link
WO (1) WO2024220980A2 (en)

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101633930B (en) * 2009-06-11 2012-11-07 陕西师范大学 Carrier for rapidly screening small interfering RNA and construction method thereof and application thereof
US20130243824A1 (en) * 2007-11-07 2013-09-19 Ono Pharmaceutical Co., Ltd. Sustained release composition containing sdf-1
US20160346281A1 (en) * 2014-01-03 2016-12-01 The Regents Of The University Of Michigan Treatment of neurological disorders
US20190062736A1 (en) * 2017-08-22 2019-02-28 Board Of Regents, The University Of Texas System In situ and in vivo analysis of chromatin interactions by biotinylated dcas9 protein
US20190161730A1 (en) * 2016-07-07 2019-05-30 Rubius Therapeutics, Inc. Compositions and methods related to therapeutic cell systems expressing exogenous rna
US20210172017A1 (en) * 2019-09-19 2021-06-10 The Broad Institute, Inc. Methods of in vivo evaluation of gene function
WO2023004409A1 (en) * 2021-07-23 2023-01-26 Beam Therapeutics Inc. Guide rnas for crispr/cas editing systems

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130243824A1 (en) * 2007-11-07 2013-09-19 Ono Pharmaceutical Co., Ltd. Sustained release composition containing sdf-1
CN101633930B (en) * 2009-06-11 2012-11-07 陕西师范大学 Carrier for rapidly screening small interfering RNA and construction method thereof and application thereof
US20160346281A1 (en) * 2014-01-03 2016-12-01 The Regents Of The University Of Michigan Treatment of neurological disorders
US20190161730A1 (en) * 2016-07-07 2019-05-30 Rubius Therapeutics, Inc. Compositions and methods related to therapeutic cell systems expressing exogenous rna
US20190062736A1 (en) * 2017-08-22 2019-02-28 Board Of Regents, The University Of Texas System In situ and in vivo analysis of chromatin interactions by biotinylated dcas9 protein
US20210172017A1 (en) * 2019-09-19 2021-06-10 The Broad Institute, Inc. Methods of in vivo evaluation of gene function
WO2023004409A1 (en) * 2021-07-23 2023-01-26 Beam Therapeutics Inc. Guide rnas for crispr/cas editing systems

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ARIMURA NARIKO, OKADA MAKO, TAYA SHINICHIRO, DEWA KEN-ICHI, TSUZUKI AKIKO, UETAKE HIROTOMO, MIYASHITA SATOSHI, HASHIZUME KOICHI, S: "DSCAM regulates delamination of neurons in the developing midbrain", SCIENCE ADVANCES, AMERICAN ASSOCIATION FOR THE ADVANCEMENT OF SCIENCE, US, vol. 6, no. 36, 4 September 2020 (2020-09-04), US , pages 1 - 14, XP093248914, ISSN: 2375-2548, DOI: 10.1126/sciadv.aba1693 *
FUERST ET AL.: "Cell autonomy of DSCAM function in retinal development", DEVELOPMENTAL BIOLOGY, vol. 361, no. 2, 29 October 2011 (2011-10-29), pages 326 - 337, XP028350558, DOI: 10.1016/j.ydbio.2011.10.028 *

Also Published As

Publication number Publication date
WO2024220980A2 (en) 2024-10-24

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