WO2021041716A3 - Therapeutic editing to treat cardiomyopathy - Google Patents

Therapeutic editing to treat cardiomyopathy Download PDF

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Publication number
WO2021041716A3
WO2021041716A3 PCT/US2020/048254 US2020048254W WO2021041716A3 WO 2021041716 A3 WO2021041716 A3 WO 2021041716A3 US 2020048254 W US2020048254 W US 2020048254W WO 2021041716 A3 WO2021041716 A3 WO 2021041716A3
Authority
WO
WIPO (PCT)
Prior art keywords
therapeutic editing
treat cardiomyopathy
cardiomyopathy
treat
editing
Prior art date
Application number
PCT/US2020/048254
Other languages
French (fr)
Other versions
WO2021041716A2 (en
Inventor
Bruce Conklin
Hana GHANIM
Original Assignee
The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glads
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 The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glads filed Critical The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glads
Publication of WO2021041716A2 publication Critical patent/WO2021041716A2/en
Publication of WO2021041716A3 publication Critical patent/WO2021041716A3/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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

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)
  • 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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Described herein are guide RNAs that can be used to treat or inhibit the onset of cardiomyopathy.
PCT/US2020/048254 2019-08-28 2020-08-27 Therapeutic editing to treat cardiomyopathy WO2021041716A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962892856P 2019-08-28 2019-08-28
US62/892,856 2019-08-28

Publications (2)

Publication Number Publication Date
WO2021041716A2 WO2021041716A2 (en) 2021-03-04
WO2021041716A3 true WO2021041716A3 (en) 2021-04-22

Family

ID=74686035

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2020/048254 WO2021041716A2 (en) 2019-08-28 2020-08-27 Therapeutic editing to treat cardiomyopathy

Country Status (1)

Country Link
WO (1) WO2021041716A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023080755A1 (en) * 2021-11-08 2023-05-11 고려대학교 산학협력단 Method for preparing stem cell-derived cardiomyopathy model cell line by using crispr-based base editing technology, and cardiomyopathy cell line prepared by same method
CN115851751A (en) * 2022-12-21 2023-03-28 百世诺（北京）医疗科技有限公司 Hypertrophic cardiomyopathy variant gene TNNT2 and application thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130029866A1 (en) * 2011-07-21 2013-01-31 Ning Sun Cardiomyocytes from induced pluripotent stem cells from patients and methods of use thereof
US20170137968A1 (en) * 2015-09-07 2017-05-18 Global Gene Corporation Pte. Ltd. Method and System for Diagnosing Disease and Generating Treatment Recommendations

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130029866A1 (en) * 2011-07-21 2013-01-31 Ning Sun Cardiomyocytes from induced pluripotent stem cells from patients and methods of use thereof
US20170137968A1 (en) * 2015-09-07 2017-05-18 Global Gene Corporation Pte. Ltd. Method and System for Diagnosing Disease and Generating Treatment Recommendations

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
TAKAMARU ISHIZU, SHUICHIRO HIGO, YUKI MASUMURA, YASUAKI KOHAMA, MIKIO SHIBA, TOMOAKI HIGO, MASATO SHIBAMOTO, AKITO NAKAGAWA, SACHI: "Targeted Genome Replacement via Homology-directed Repair in Non-dividing Cardiomyocytes", SCIENTIFIC REPORTS, vol. 7, no. 9363, 24 August 2017 (2017-08-24), pages 1 - 11, XP055693490 *

Also Published As

Publication number Publication date
WO2021041716A2 (en) 2021-03-04

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