HK1250720A1 - 使用外顯子跳躍改善2c型肢帶型肌營養不良症的組合物和方法 - Google Patents

使用外顯子跳躍改善2c型肢帶型肌營養不良症的組合物和方法

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Publication number
HK1250720A1
HK1250720A1 HK18110160.3A HK18110160A HK1250720A1 HK 1250720 A1 HK1250720 A1 HK 1250720A1 HK 18110160 A HK18110160 A HK 18110160A HK 1250720 A1 HK1250720 A1 HK 1250720A1
Authority
HK
Hong Kong
Prior art keywords
compositions
methods
muscular dystrophy
exon skipping
girdle muscular
Prior art date
Application number
HK18110160.3A
Other languages
English (en)
Inventor
Elizabeth Mcnally
Eugene Wyatt
Original Assignee
Univ Chicago
Univ Northwestern
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 Univ Chicago, Univ Northwestern filed Critical Univ Chicago
Publication of HK1250720A1 publication Critical patent/HK1250720A1/zh

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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
    • C12N15/1138—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 receptors or cell surface proteins
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Footwear And Its Accessory, Manufacturing Method And Apparatuses (AREA)
HK18110160.3A 2015-04-08 2018-08-07 使用外顯子跳躍改善2c型肢帶型肌營養不良症的組合物和方法 HK1250720A1 (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562144712P 2015-04-08 2015-04-08
PCT/US2016/026477 WO2016164602A1 (en) 2015-04-08 2016-04-07 Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping

Publications (1)

Publication Number Publication Date
HK1250720A1 true HK1250720A1 (zh) 2019-01-11

Family

ID=57073329

Family Applications (1)

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HK18110160.3A HK1250720A1 (zh) 2015-04-08 2018-08-07 使用外顯子跳躍改善2c型肢帶型肌營養不良症的組合物和方法

Country Status (17)

Country Link
US (5) US10273483B2 (zh)
EP (1) EP3283500B1 (zh)
BR (1) BR112017021485A2 (zh)
CA (1) CA2981960C (zh)
CY (1) CY1123599T1 (zh)
DK (1) DK3283500T3 (zh)
ES (1) ES2846902T3 (zh)
HK (1) HK1250720A1 (zh)
HR (1) HRP20210139T1 (zh)
HU (1) HUE052604T2 (zh)
LT (1) LT3283500T (zh)
PL (1) PL3283500T3 (zh)
PT (1) PT3283500T (zh)
RS (1) RS61349B1 (zh)
SI (1) SI3283500T1 (zh)
TN (1) TN2017000427A1 (zh)
WO (1) WO2016164602A1 (zh)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9499817B2 (en) * 2012-09-06 2016-11-22 The University Of Chicago Antisense polynucleotides to induce exon skipping and methods of treating dystrophies
SI3283500T1 (sl) * 2015-04-08 2020-12-31 The University Of Chicago Sestavki in postopki za popravilo medenično ramenske distrofije tipa 2C z uporabo preskakovanja eksonov

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SI3283500T1 (sl) * 2015-04-08 2020-12-31 The University Of Chicago Sestavki in postopki za popravilo medenično ramenske distrofije tipa 2C z uporabo preskakovanja eksonov

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TN2017000427A1 (en) 2019-04-12
PT3283500T (pt) 2021-01-28
HRP20210139T1 (hr) 2021-03-19
US20230167452A1 (en) 2023-06-01
BR112017021485A2 (pt) 2018-07-03
DK3283500T3 (en) 2020-11-16
EP3283500A1 (en) 2018-02-21
WO2016164602A1 (en) 2016-10-13
US20210032631A1 (en) 2021-02-04
EP3283500B1 (en) 2020-11-11
US10273483B2 (en) 2019-04-30
US20180080030A1 (en) 2018-03-22
RS61349B1 (sr) 2021-02-26
CA2981960C (en) 2023-09-19
US10801029B2 (en) 2020-10-13
LT3283500T (lt) 2020-12-10
US20190249180A1 (en) 2019-08-15
CY1123599T1 (el) 2022-03-24
PL3283500T3 (pl) 2021-05-31
EP3283500A4 (en) 2019-01-23
ES2846902T3 (es) 2021-07-30
CA2981960A1 (en) 2016-10-13
US20220119820A1 (en) 2022-04-21
SI3283500T1 (sl) 2020-12-31
HUE052604T2 (hu) 2021-05-28

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