AU2016358172B2 - Materials and methods for treatment of titin-based myopathies and other titinopaties - Google Patents

Materials and methods for treatment of titin-based myopathies and other titinopaties Download PDF

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AU2016358172B2
AU2016358172B2 AU2016358172A AU2016358172A AU2016358172B2 AU 2016358172 B2 AU2016358172 B2 AU 2016358172B2 AU 2016358172 A AU2016358172 A AU 2016358172A AU 2016358172 A AU2016358172 A AU 2016358172A AU 2016358172 B2 AU2016358172 B2 AU 2016358172B2
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nucleic acid
sequence
cell
dna
titin
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AU2016358172A1 (en
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Rachael POTTER
Louise RODINO-KLAPAC
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Nationwide Childrens Hospital Inc
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    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/39—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
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    • 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
    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2750/14011—Parvoviridae
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
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AU2016358172A 2015-11-16 2016-11-15 Materials and methods for treatment of titin-based myopathies and other titinopaties Active AU2016358172B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562255887P 2015-11-16 2015-11-16
US62/255,887 2015-11-16
PCT/US2016/062052 WO2017087395A1 (en) 2015-11-16 2016-11-15 Materials and methods for treatment of titin-based myopathies and other titinopaties

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AU2016358172A1 AU2016358172A1 (en) 2018-06-07
AU2016358172B2 true AU2016358172B2 (en) 2023-06-22

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US (2) US11617783B2 (https=)
EP (1) EP3377042A4 (https=)
JP (3) JP7418957B2 (https=)
KR (1) KR102877920B1 (https=)
CN (1) CN108463211B (https=)
AU (1) AU2016358172B2 (https=)
CA (1) CA3005633C (https=)
CO (1) CO2018006133A2 (https=)
EA (1) EA201891192A1 (https=)
HK (1) HK1257955A1 (https=)
IL (1) IL259441B2 (https=)
MX (1) MX2018006116A (https=)
SG (1) SG11201804166YA (https=)
WO (1) WO2017087395A1 (https=)

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