CA3059213A1 - Methods of treating amyotrophic lateral sclerosis (als) - Google Patents

Methods of treating amyotrophic lateral sclerosis (als) Download PDF

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Publication number
CA3059213A1
CA3059213A1 CA3059213A CA3059213A CA3059213A1 CA 3059213 A1 CA3059213 A1 CA 3059213A1 CA 3059213 A CA3059213 A CA 3059213A CA 3059213 A CA3059213 A CA 3059213A CA 3059213 A1 CA3059213 A1 CA 3059213A1
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Prior art keywords
protein
grna
cell
gene editing
nucleic acid
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CA3059213A
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English (en)
French (fr)
Inventor
Christian Mueller
Abbas Abdallah
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University of Massachusetts Amherst
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University of Massachusetts Amherst
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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
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    • A01K2227/10—Mammal
    • A01K2227/105—Murine
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
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    • 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 [CRISPR]
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Biodiversity & Conservation Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3059213A 2017-05-09 2018-05-09 Methods of treating amyotrophic lateral sclerosis (als) Pending CA3059213A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762503909P 2017-05-09 2017-05-09
US62/503,909 2017-05-09
PCT/US2018/031880 WO2018208972A1 (en) 2017-05-09 2018-05-09 Methods of treating amyotrophic lateral sclerosis (als)

Publications (1)

Publication Number Publication Date
CA3059213A1 true CA3059213A1 (en) 2018-11-15

Family

ID=64104908

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CA3059213A Pending CA3059213A1 (en) 2017-05-09 2018-05-09 Methods of treating amyotrophic lateral sclerosis (als)

Country Status (6)

Country Link
US (2) US11859179B2 (https=)
EP (1) EP3622073A4 (https=)
JP (1) JP7327803B2 (https=)
AU (1) AU2018264996A1 (https=)
CA (1) CA3059213A1 (https=)
WO (1) WO2018208972A1 (https=)

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Publication number Priority date Publication date Assignee Title
CA2942515C (en) 2014-03-18 2025-12-09 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
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CN109862785B (zh) 2016-09-30 2022-09-06 瑞泽恩制药公司 C9orf72基因座中具有六核苷酸重复扩增的非人类动物
US11859179B2 (en) 2017-05-09 2024-01-02 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (ALS)
CN111448321A (zh) 2017-09-22 2020-07-24 马萨诸塞大学 Sod1双表达载体及其用途
KR102925054B1 (ko) 2018-12-20 2026-02-10 리제너론 파마슈티칼스 인코포레이티드 뉴클레아제-매개 반복부 팽창
TW202521561A (zh) * 2019-04-23 2025-06-01 美商聖加莫治療股份有限公司 染色體9開放讀框72基因表現之調節子及其用途
AU2021237633A1 (en) * 2020-03-18 2022-10-06 Scribe Therapeutics Inc. Compositions and methods for the targeting of C9orf72
EP4222264A1 (en) * 2020-09-30 2023-08-09 CRISPR Therapeutics AG Materials and methods for treatment of amyotrophic lateral sclerosis
GB202105455D0 (en) * 2021-04-16 2021-06-02 Ucl Business Ltd Composition
WO2023025103A1 (en) * 2021-08-22 2023-03-02 Huigene Therapeutics Co., Ltd. Crispr-cas13 system for treating sod1-associated diseases
EP4430188A1 (en) * 2021-11-09 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of amyotrophic lateral sclerosis

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