PL2548560T3 - Kompozycje i sposoby modulowania splicingu SMN2 - Google Patents

Kompozycje i sposoby modulowania splicingu SMN2

Info

Publication number
PL2548560T3
PL2548560T3 PL12188625T PL12188625T PL2548560T3 PL 2548560 T3 PL2548560 T3 PL 2548560T3 PL 12188625 T PL12188625 T PL 12188625T PL 12188625 T PL12188625 T PL 12188625T PL 2548560 T3 PL2548560 T3 PL 2548560T3
Authority
PL
Poland
Prior art keywords
modulation
compositions
methods
smn2 splicing
smn2
Prior art date
Application number
PL12188625T
Other languages
English (en)
Inventor
Brenda F Baker
Adrian R Krainer
Yimin Hua
Original Assignee
Biogen Ma Inc
Cold Spring Harbor Laboratory
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 Biogen Ma Inc, Cold Spring Harbor Laboratory filed Critical Biogen Ma Inc
Publication of PL2548560T3 publication Critical patent/PL2548560T3/pl

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
PL12188625T 2005-06-23 2006-06-23 Kompozycje i sposoby modulowania splicingu SMN2 PL2548560T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US69354205P 2005-06-23 2005-06-23
EP06773838A EP1910395B1 (en) 2005-06-23 2006-06-23 Compositions and methods for modulation of SMN2 splicing
EP12188625.3A EP2548560B1 (en) 2005-06-23 2006-06-23 Compositions and methods for modulation of SMN2 splicing

Publications (1)

Publication Number Publication Date
PL2548560T3 true PL2548560T3 (pl) 2015-11-30

Family

ID=37595872

Family Applications (3)

Application Number Title Priority Date Filing Date
PL12188625T PL2548560T3 (pl) 2005-06-23 2006-06-23 Kompozycje i sposoby modulowania splicingu SMN2
PL06773838T PL1910395T3 (pl) 2005-06-23 2006-06-23 Kompozycja i sposób modulacji splicingu SMN2
PL17185196T PL3308788T3 (pl) 2005-06-23 2006-06-23 Kompozycje i sposoby modulacji splicingu smn2

Family Applications After (2)

Application Number Title Priority Date Filing Date
PL06773838T PL1910395T3 (pl) 2005-06-23 2006-06-23 Kompozycja i sposób modulacji splicingu SMN2
PL17185196T PL3308788T3 (pl) 2005-06-23 2006-06-23 Kompozycje i sposoby modulacji splicingu smn2

Country Status (13)

Country Link
US (6) US8361977B2 (pl)
EP (5) EP2644700A1 (pl)
CY (2) CY2017040I2 (pl)
DK (3) DK2548560T3 (pl)
ES (3) ES2545223T3 (pl)
HK (2) HK1181307A1 (pl)
HU (2) HUE027486T2 (pl)
LT (2) LT3308788T (pl)
LU (1) LUC00046I2 (pl)
PL (3) PL2548560T3 (pl)
PT (3) PT3308788T (pl)
SI (2) SI3308788T1 (pl)
WO (1) WO2007002390A2 (pl)

Families Citing this family (97)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
PL2548560T3 (pl) 2005-06-23 2015-11-30 Biogen Ma Inc Kompozycje i sposoby modulowania splicingu SMN2
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
CN101896186A (zh) 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
EP2219680A2 (en) 2007-11-13 2010-08-25 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
WO2009151546A2 (en) * 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
US8932818B2 (en) 2008-08-13 2015-01-13 Ptc Therapeutics, Inc. Screening assays for compounds that modulate programmed ribosomal frameshifting
ES2616051T3 (es) 2008-12-02 2017-06-09 Wave Life Sciences Japan, Inc. Método para la síntesis de ácidos nucleicos modificados en el átomo de fósforo
IT1398361B1 (it) * 2009-01-21 2013-02-22 Fond Santa Lucia Uso di mutanti dominanti negativi di sam68 per il trattamento della sma
CN102625840A (zh) * 2009-04-10 2012-08-01 肌肉学研究协会 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法
WO2010120820A1 (en) * 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
PT3449926T (pt) * 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito
US9744183B2 (en) 2009-07-06 2017-08-29 Wave Life Sciences Ltd. Nucleic acid prodrugs and methods of use thereof
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
TWI620756B (zh) 2010-05-28 2018-04-11 薩羅塔治療公司 具有經修飾之單元間連結及／或末端基團之寡核苷酸類似物
EP2582397A4 (en) * 2010-06-15 2014-10-29 Isis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR MODULATING THE INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
WO2012031243A2 (en) 2010-09-03 2012-03-08 Avi Biopharma, Inc. dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
IT1405793B1 (it) * 2010-10-15 2014-01-24 Univ Ferrara Molecola di u1snrna umano modificato, gene codificante per la molecola di u1snrna umano modificato, vettore di espressione includente il gene, e loro uso in terapia genica
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
WO2012065143A1 (en) 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
US20140128449A1 (en) * 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
IL273838B (en) 2011-05-05 2022-09-01 Sarepta Therapeutics Inc Peptide and oligonucleotide conjugates, preparations containing them and their uses
EP2723864A2 (en) * 2011-06-23 2014-04-30 Cold Spring Harbor Laboratory Phenocopy model of disease
KR20140068884A (ko) 2011-07-19 2014-06-09 웨이브 라이프 사이언시스 피티이. 리미티드 관능화된 핵산의 합성 방법
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
CA2855241A1 (en) 2011-11-11 2013-05-16 Santaris Pharma A/S Compounds for the modulation of smn2 splicing
WO2013074834A1 (en) 2011-11-18 2013-05-23 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
US10066228B2 (en) 2011-11-30 2018-09-04 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
CA2857664A1 (en) 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides targeting within the smn2 pre-mrna for use ininduced exon inclusion in spinal muscle atrophy
US9725716B2 (en) 2011-12-06 2017-08-08 Ohio State Innovation Foundation and Research Institute at Nationwide Children's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
WO2013112053A1 (en) 2012-01-27 2013-08-01 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
WO2013173645A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating utrn expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EP2850190B1 (en) 2012-05-16 2020-07-08 Translate Bio MA, Inc. Compositions and methods for modulating mecp2 expression
KR20150030205A (ko) * 2012-05-16 2015-03-19 라나 테라퓨틱스, 인크. Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법
CN112007045A (zh) 2012-07-13 2020-12-01 波涛生命科学有限公司 手性控制
JP6268157B2 (ja) 2012-07-13 2018-01-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ 不斉補助基
US9926559B2 (en) 2013-01-09 2018-03-27 Biogen Ma Inc. Compositions and methods for modulation of SMN2 splicing in a subject
WO2014113540A1 (en) * 2013-01-16 2014-07-24 Iowa State University Research Foundation, Inc. A deep intronic target for splicing correction on spinal muscular atrophy gene
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
TWI736514B (zh) 2013-09-05 2021-08-21 美商薩羅塔治療公司 酸性α葡萄糖苷酶之反義股誘導之外顯子２包含
JP2016534035A (ja) 2013-10-04 2016-11-04 ラナ セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症を治療するための組成物及び方法
WO2015108048A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
PL3094728T3 (pl) 2014-01-16 2022-07-04 Wave Life Sciences Ltd. Projekt chiralny
US9845469B2 (en) 2014-02-10 2017-12-19 Ohio State Innovation Foundation Antisense oligonucleotides for treatment of spinal muscular atrophy
WO2015161170A2 (en) 2014-04-17 2015-10-22 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
US10858650B2 (en) 2014-10-30 2020-12-08 The General Hospital Corporation Methods for modulating ATRX-dependent gene repression
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
WO2016164896A2 (en) 2015-04-10 2016-10-13 Ionis Pharmaceuticals, Inc. Modulation of smn expression
JP6873052B2 (ja) 2015-06-01 2021-05-19 サレプタ セラピューティクス，インコーポレイテッド Ｖｉｉ型コラーゲンにおけるアンチセンス誘導エクソン排除
WO2016196897A1 (en) 2015-06-04 2016-12-08 Sarepta Therapeutics, Inc. Methods and compounds for treatment of lymphocyte-related diseases and conditions
AU2016317667A1 (en) 2015-08-28 2018-03-22 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
EP3858993A1 (en) 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
US9669109B1 (en) 2015-11-24 2017-06-06 Universita Di Ferrara Modified human U1snRNA molecule, a gene encoding for the modified human U1snRNA molecule, an expression vector including the gene, and the use thereof in gene therapy of familial dysautonomia and spinal muscular atrophy
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
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
WO2017136435A1 (en) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Compounds for modulating fc-epsilon-ri-beta expression and uses thereof
AU2017254106B2 (en) 2016-04-18 2024-07-11 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
JP7075359B2 (ja) 2016-06-14 2022-05-25 バイオジェン・エムエイ・インコーポレイテッド オリゴヌクレオチドの精製のための疎水性相互作用クロマトグラフィー
EP3471779A4 (en) 2016-06-16 2020-07-08 Ionis Pharmaceuticals, Inc. COMBINATIONS FOR MODULATING THE SMN EXPRESSION
CN109689668B (zh) 2016-06-24 2023-02-17 比奥根Ma公司 无加帽步骤的硫醇化寡核苷酸的合成
US11655470B2 (en) 2016-07-05 2023-05-23 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Diagnosing COL6-related disorders and methods for treating same
EP3485016A4 (en) * 2016-07-15 2020-03-18 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING TRANSCRIPTION PROCESSING
EP4035659A1 (en) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes for delivery of therapeutic agents
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
SI3589730T1 (sl) 2017-02-28 2024-04-30 The Trustees Of The University Of Pennsylvania Adeno-povezan virus (AAV) clade F vektor in njihova uporaba
WO2018195338A1 (en) 2017-04-20 2018-10-25 Atyr Pharma, Inc. Compositions and methods for treating lung inflammation
US11549125B2 (en) 2017-08-09 2023-01-10 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Closed-ended, linear, duplex adenoassociated virus DNA, and uses thereof
FI3673080T3 (fi) 2017-08-25 2023-11-23 Stoke Therapeutics Inc Antisense-oligomeerejä häiriötilojen ja sairauksien hoitoon
SG11202001783YA (en) 2017-10-12 2020-03-30 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
JP7394753B2 (ja) 2017-10-18 2023-12-08 サレプタ セラピューティクス， インコーポレイテッド アンチセンスオリゴマー化合物
SG11202004406VA (en) 2017-11-08 2020-06-29 Avexis Inc Means and method for preparing viral vectors and uses of same
JP7455746B2 (ja) 2018-01-12 2024-03-26 ブリストル－マイヤーズ スクイブ カンパニー アルファ－シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用
MX2020007852A (es) 2018-01-25 2021-01-29 Biogen Ma Inc Metodos para el tratamiento de la atrofia muscular espinal.
MX2020011695A (es) * 2018-05-04 2021-02-26 Stoke Therapeutics Inc Métodos y composiciones para el tratamiento de la enfermedad por almacenamiento de éster de colesterilo.
CN112601557A (zh) 2018-06-08 2021-04-02 诺华股份有限公司 用于测量药物产品效力的基于细胞的测定
EP3873934A2 (en) 2018-10-29 2021-09-08 Biogen MA Inc. Humanized and stabilized fc5 variants for enhancement of blood brain barrier transport
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
US20220001028A1 (en) 2018-11-30 2022-01-06 Novartis Ag Aav viral vectors and uses thereof
CA3162618A1 (en) 2019-03-20 2020-09-24 Peter Jungsoo PARK Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
JP2022531876A (ja) 2019-05-08 2022-07-12 バイオジェン・エムエイ・インコーポレイテッド オリゴヌクレオチドの収束液相合成
PL3999643T3 (pl) 2019-07-19 2024-02-19 Biogen Ma Inc. Sposoby leczenia rdzeniowego zaniku mięśni i zapobiegania mu
CN115279379A (zh) * 2020-02-28 2022-11-01 Ionis 制药公司 用于调节smn2的化合物和方法
MX2022014151A (es) 2020-05-11 2022-11-30 Stoke Therapeutics Inc Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades.
KR20240012425A (ko) 2021-05-10 2024-01-29 엔트라다 테라퓨틱스, 인크. 세포내 치료제를 위한 조성물 및 방법
WO2023073526A1 (en) 2021-10-25 2023-05-04 Novartis Ag Methods for improving adeno-associated virus (aav) delivery
FR3128873A1 (fr) 2021-11-10 2023-05-12 Biophytis Phytoecdysones et/ou dérivés de 20-hydroxyecdysone en combinaison avec un principe actif visant à restaurer l’expression SMN pour leur utilisation dans le traitement de l’amyotrophie spinale
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5176996A (en) * 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
FR2645345A1 (fr) * 1989-03-31 1990-10-05 Thomson Csf Procede de modulation dirigee de la composition ou du dopage de semi-conducteurs, notamment pour la realisation de composants electroniques monolithiques de type planar, utilisation et produits correspondants
US5256775A (en) * 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US6582908B2 (en) * 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
FR2692265B1 (fr) 1992-05-25 1996-11-08 Centre Nat Rech Scient Composes biologiquement actifs de type phosphotriesters.
JPH08510130A (ja) * 1993-05-11 1996-10-29 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法
FR2705099B1 (fr) 1993-05-12 1995-08-04 Centre Nat Rech Scient Oligonucléotides phosphorothioates triesters et procédé de préparation.
US5801154A (en) * 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
NZ282981A (en) 1994-02-28 1998-08-26 Agritech Lab Inc Increasing egg production in female birds by administering heterologous inhibin protein and a carrier protein
US7034009B2 (en) * 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US20030228597A1 (en) * 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6214986B1 (en) * 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
DE19906484A1 (de) * 1999-02-17 2000-09-07 Wegerer Kristina Elektrische Maschine
DE60033927T2 (de) * 1999-05-04 2007-11-29 Santaris Pharma A/S L-ribo-lna analoge
US6649411B2 (en) 1999-07-30 2003-11-18 The United States Of America As Represented By The Department Of Health And Human Services Methods of inhibiting cancer cells with ADNF III antisense oligonucleotides
US6770633B1 (en) 1999-10-26 2004-08-03 Immusol, Inc. Ribozyme therapy for the treatment of proliferative skin and eye diseases
US6962906B2 (en) * 2000-03-14 2005-11-08 Active Motif Oligonucleotide analogues, methods of synthesis and methods of use
AU2002232536A1 (en) 2000-11-09 2002-05-21 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
US6376508B1 (en) * 2000-12-13 2002-04-23 Academia Sinica Treatments for spinal muscular atrophy
GB0219143D0 (en) * 2002-08-16 2002-09-25 Univ Leicester Modified tailed oligonucleotides
EP2278005A3 (en) * 2002-11-14 2012-05-23 Dharmacon, Inc. Fuctional and hyperfunctional sirna
WO2004113867A2 (en) 2003-06-16 2004-12-29 University Of Massachusetts Exon analysis
GB0425625D0 (en) * 2004-11-22 2004-12-22 Royal College Of Surgeons Ie Treatment of disease
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
PL2548560T3 (pl) 2005-06-23 2015-11-30 Biogen Ma Inc Kompozycje i sposoby modulowania splicingu SMN2
WO2010091308A2 (en) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
WO2010120820A1 (en) * 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
PT3449926T (pt) * 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito

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