AU2019347517A1 - Compositions and methods for lactate dehydrogenase (LDHA) gene editing - Google Patents

Compositions and methods for lactate dehydrogenase (LDHA) gene editing Download PDF

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Publication number
AU2019347517A1
AU2019347517A1 AU2019347517A AU2019347517A AU2019347517A1 AU 2019347517 A1 AU2019347517 A1 AU 2019347517A1 AU 2019347517 A AU2019347517 A AU 2019347517A AU 2019347517 A AU2019347517 A AU 2019347517A AU 2019347517 A1 AU2019347517 A1 AU 2019347517A1
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seq
composition
nos
guide
sequence selected
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Zachary William DYMEK
Anette HUEBNER
Bradley Andrew MURRAY
Shobu ODATE
Srijani SRIDHAR
Walter Strapps
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Intellia Therapeutics Inc
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Intellia Therapeutics Inc
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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
    • 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
    • C12N15/1137—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 enzymes
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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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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
    • C12Y101/01027—L-Lactate dehydrogenase (1.1.1.27)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

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AU2019347517A 2018-09-28 2019-09-27 Compositions and methods for lactate dehydrogenase (LDHA) gene editing Withdrawn AU2019347517A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862738956P 2018-09-28 2018-09-28
US62/738,956 2018-09-28
US201962834334P 2019-04-15 2019-04-15
US62/834,334 2019-04-15
US201962841740P 2019-05-01 2019-05-01
US62/841,740 2019-05-01
PCT/US2019/053423 WO2020069296A1 (en) 2018-09-28 2019-09-27 Compositions and methods for lactate dehydrogenase (ldha) gene editing

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AU2019347517A1 true AU2019347517A1 (en) 2021-05-06

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AU2019347517A Withdrawn AU2019347517A1 (en) 2018-09-28 2019-09-27 Compositions and methods for lactate dehydrogenase (LDHA) gene editing

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US (2) US20210222173A1 (https=)
EP (1) EP3856901A1 (https=)
JP (1) JP2022502055A (https=)
KR (1) KR20210086621A (https=)
CN (1) CN113056559A (https=)
AU (1) AU2019347517A1 (https=)
BR (1) BR112021005718A2 (https=)
CA (1) CA3114425A1 (https=)
CO (1) CO2021005231A2 (https=)
IL (1) IL281529A (https=)
MX (1) MX2021003457A (https=)
PH (1) PH12021550686A1 (https=)
SG (1) SG11202102660RA (https=)
TW (1) TW202028460A (https=)
WO (1) WO2020069296A1 (https=)

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SG11202102660RA (en) 2021-04-29
JP2022502055A (ja) 2022-01-11
TW202028460A (zh) 2020-08-01
KR20210086621A (ko) 2021-07-08
CA3114425A1 (en) 2020-04-02
CO2021005231A2 (es) 2021-06-10
MX2021003457A (es) 2021-07-16
PH12021550686A1 (en) 2022-02-28
CN113056559A (zh) 2021-06-29
EP3856901A1 (en) 2021-08-04
BR112021005718A2 (pt) 2021-06-22
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