AU2018264996A1 - Methods of treating Amyotrophic Lateral Sclerosis (ALS) - Google Patents

Methods of treating Amyotrophic Lateral Sclerosis (ALS) Download PDF

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Publication number
AU2018264996A1
AU2018264996A1 AU2018264996A AU2018264996A AU2018264996A1 AU 2018264996 A1 AU2018264996 A1 AU 2018264996A1 AU 2018264996 A AU2018264996 A AU 2018264996A AU 2018264996 A AU2018264996 A AU 2018264996A AU 2018264996 A1 AU2018264996 A1 AU 2018264996A1
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Prior art keywords
protein
grna
cell
gene editing
nucleic acid
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AU2018264996A
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Abbas Abdallah
Christian Mueller
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University of Massachusetts Amherst
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University of Massachusetts Amherst
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    • 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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    • 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
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    • A01K2267/03—Animal model, e.g. for test or diseases
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    • 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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    • C12N2750/00011—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
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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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  • 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)
AU2018264996A 2017-05-09 2018-05-09 Methods of treating Amyotrophic Lateral Sclerosis (ALS) Abandoned AU2018264996A1 (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)

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AU2018264996A1 true AU2018264996A1 (en) 2019-12-05

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AU2018264996A Abandoned AU2018264996A1 (en) 2017-05-09 2018-05-09 Methods of treating Amyotrophic Lateral Sclerosis (ALS)

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US (2) US11859179B2 (enExample)
EP (1) EP3622073A4 (enExample)
JP (1) JP7327803B2 (enExample)
AU (1) AU2018264996A1 (enExample)
CA (1) CA3059213A1 (enExample)
WO (1) WO2018208972A1 (enExample)

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WO2019060686A1 (en) 2017-09-22 2019-03-28 University Of Massachusetts NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF
JP7449291B2 (ja) 2018-12-20 2024-03-13 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ヌクレアーゼ媒介リピート伸長
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GB202105455D0 (en) * 2021-04-16 2021-06-02 Ucl Business Ltd Composition
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