CA2963288A1 - Targeted augmentation of nuclear gene output - Google Patents

Targeted augmentation of nuclear gene output Download PDF

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Publication number
CA2963288A1
CA2963288A1 CA2963288A CA2963288A CA2963288A1 CA 2963288 A1 CA2963288 A1 CA 2963288A1 CA 2963288 A CA2963288 A CA 2963288A CA 2963288 A CA2963288 A CA 2963288A CA 2963288 A1 CA2963288 A1 CA 2963288A1
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Prior art keywords
mrna
fold
nucleobases
intron
target protein
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CA2963288A
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English (en)
French (fr)
Inventor
Adrian Krainer
Isabel AZNAREZ
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Cold Spring Harbor Laboratory
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Cold Spring Harbor Laboratory
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Publication of CA2963288A1 publication Critical patent/CA2963288A1/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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CA2963288A 2014-10-03 2015-10-03 Targeted augmentation of nuclear gene output Pending CA2963288A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462059847P 2014-10-03 2014-10-03
US62/059,847 2014-10-03
PCT/US2015/053896 WO2016054615A2 (en) 2014-10-03 2015-10-03 Targeted augmentation of nuclear gene output

Publications (1)

Publication Number Publication Date
CA2963288A1 true CA2963288A1 (en) 2016-04-07

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CA2963288A Pending CA2963288A1 (en) 2014-10-03 2015-10-03 Targeted augmentation of nuclear gene output

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US (4) US9976143B2 (enExample)
EP (1) EP3201339A4 (enExample)
JP (1) JP6867945B2 (enExample)
KR (1) KR102620328B1 (enExample)
CN (1) CN107109411B (enExample)
AU (1) AU2015327836B2 (enExample)
CA (1) CA2963288A1 (enExample)
IL (1) IL251470B (enExample)
SG (1) SG11201702682PA (enExample)
WO (1) WO2016054615A2 (enExample)

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WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
US10525076B2 (en) 2015-02-20 2020-01-07 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
US9840709B2 (en) 2015-02-20 2017-12-12 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
EP3390666A4 (en) * 2015-12-14 2019-08-07 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017106211A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of polycystic kidney disease
EP3390636B1 (en) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Antisense oligomers for treatment of dravet syndrome
JP7051683B2 (ja) * 2015-12-14 2022-04-11 コールド スプリング ハーバー ラボラトリー 結節性硬化症の処置のためのアンチセンスオリゴマー
JP7036723B2 (ja) * 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 眼疾患の処置のための組成物および方法
AU2018218280B2 (en) 2017-02-07 2024-10-17 The Regents Of The University Of California Gene therapy for haploinsufficiency
EP3673080B1 (en) 2017-08-25 2023-10-18 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
EA202190581A1 (ru) 2018-08-20 2021-07-13 Рогкон, Инк. Антисмысловые олигонуклеотиды, нацеленные на scn2a, для лечения энцефалопатии scn1a
WO2020154462A1 (en) * 2019-01-23 2020-07-30 Rogcon U.R., Inc. Antisense oligonucleotides targeting scn2a retained introns
CN113748209A (zh) 2019-02-27 2021-12-03 斯托克制药公司 用于治疗病况和疾病的反义寡聚体
EP3942049A4 (en) 2019-03-20 2023-10-18 President And Fellows Of Harvard College ANTISENSE OLIGONUCLEOTIDE BASED PROGRANULIN AUGMENTATION THERAPY FOR NEURODEGENERATIVE DISEASES
AU2020271530B2 (en) * 2019-04-09 2026-02-05 Envisagenics, Inc. Cancer-specific molecules and methods of use thereof
KR20220019676A (ko) * 2019-04-26 2022-02-17 스톡 테라퓨틱스, 인크. 선택적 인트론의 스플라이싱을 조절하기 위한 방법 및 조성물
CA3132838A1 (en) 2019-05-05 2020-11-12 Bat Sheva KEREM Restoration of the cftr function by splicing modulation
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