SG11201702682PA - Targeted augmentation of nuclear gene output - Google Patents

Targeted augmentation of nuclear gene output

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Publication number
SG11201702682PA
SG11201702682PA SG11201702682PA SG11201702682PA SG11201702682PA SG 11201702682P A SG11201702682P A SG 11201702682PA SG 11201702682P A SG11201702682P A SG 11201702682PA SG 11201702682P A SG11201702682P A SG 11201702682PA SG 11201702682P A SG11201702682P A SG 11201702682PA
Authority
SG
Singapore
Prior art keywords
nuclear gene
gene output
targeted augmentation
augmentation
targeted
Prior art date
Application number
SG11201702682PA
Inventor
Adrian Krainer
Isabel Aznarez
Original Assignee
Cold Spring Harbor Lab
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 Cold Spring Harbor Lab filed Critical Cold Spring Harbor Lab
Publication of SG11201702682PA publication Critical patent/SG11201702682PA/en

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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
    • 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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SG11201702682PA 2014-10-03 2015-10-03 Targeted augmentation of nuclear gene output SG11201702682PA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462059847P 2014-10-03 2014-10-03
PCT/US2015/053896 WO2016054615A2 (en) 2014-10-03 2015-10-03 Targeted augmentation of nuclear gene output

Publications (1)

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SG11201702682PA true SG11201702682PA (en) 2017-04-27

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SG11201702682PA SG11201702682PA (en) 2014-10-03 2015-10-03 Targeted augmentation of nuclear gene output

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US (3) US9976143B2 (en)
EP (1) EP3201339A4 (en)
JP (1) JP6867945B2 (en)
KR (1) KR102620328B1 (en)
CN (1) CN107109411B (en)
AU (1) AU2015327836B2 (en)
CA (1) CA2963288A1 (en)
IL (1) IL251470B (en)
SG (1) SG11201702682PA (en)
WO (1) WO2016054615A2 (en)

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US9976143B2 (en) 2014-10-03 2018-05-22 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
EP3259356B1 (en) 2015-02-20 2021-12-01 Rosalind Franklin University of Medicine and Science Antisense compounds targeting genes associated with cystic fibrosis
US10525076B2 (en) 2015-02-20 2020-01-07 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
CA3005131A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of tuberous sclerosis complex
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JP2018538287A (en) * 2015-12-14 2018-12-27 コールド スプリング ハーバー ラボラトリー Antisense oligomers for the treatment of polycystic kidney disease
CA3005249A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of kidney diseases
JP7036723B2 (en) * 2015-12-14 2022-03-15 コールド スプリング ハーバー ラボラトリー Compositions and Methods for the Treatment of Eye Diseases
CN111278991B (en) 2017-08-25 2022-04-01 斯托克制药公司 Antisense oligomers for the treatment of conditions and diseases
EP3802829A4 (en) * 2018-06-08 2022-10-19 University of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
CN113329739A (en) 2018-08-20 2021-08-31 罗贡股份有限公司 Antisense oligonucleotides targeting SCN2A for the treatment of SCN1A encephalopathy
US20220090087A1 (en) * 2019-01-23 2022-03-24 The Florey Institute Of Neuroscience And Mental Health Antisense oligonucleotides targeting scn2a retained introns
EP3942049A4 (en) 2019-03-20 2023-10-18 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
WO2020210537A1 (en) * 2019-04-09 2020-10-15 Envisagenics, Inc. Cancer-specific molecules and methods of use thereof
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US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
JP2022544702A (en) * 2019-08-19 2022-10-20 ストーク セラピューティクス，インク． Compositions and methods for modulating splicing and protein expression
WO2021102341A2 (en) * 2019-11-22 2021-05-27 Ionis Pharmaceuticals, Inc. Compounds for modulating beta globin expression
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CN113584038B (en) * 2021-09-09 2023-11-14 深圳雅济科技有限公司 Antisense oligonucleotide combination for treating retina diseases and application thereof

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