HK1181307A1 - Compositions and methods for modulation of smn2 splicing smn2 - Google Patents

Compositions and methods for modulation of smn2 splicing smn2

Info

Publication number
HK1181307A1
HK1181307A1 HK13108604.6A HK13108604A HK1181307A1 HK 1181307 A1 HK1181307 A1 HK 1181307A1 HK 13108604 A HK13108604 A HK 13108604A HK 1181307 A1 HK1181307 A1 HK 1181307A1
Authority
HK
Hong Kong
Prior art keywords
smn2
modulation
compositions
methods
splicing
Prior art date
Application number
HK13108604.6A
Other languages
English (en)
Chinese (zh)
Inventor
Brenda F Baker
Adrian R Krainer
Yimin Hua
Original Assignee
Isis Pharmaceuticals Inc
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 Isis Pharmaceuticals Inc filed Critical Isis Pharmaceuticals Inc
Publication of HK1181307A1 publication Critical patent/HK1181307A1/xx

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    • 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

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  • 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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (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)
HK13108604.6A 2005-06-23 2013-07-23 Compositions and methods for modulation of smn2 splicing smn2 HK1181307A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US69354205P 2005-06-23 2005-06-23

Publications (1)

Publication Number Publication Date
HK1181307A1 true HK1181307A1 (en) 2013-11-08

Family

ID=37595872

Family Applications (2)

Application Number Title Priority Date Filing Date
HK13108604.6A HK1181307A1 (en) 2005-06-23 2013-07-23 Compositions and methods for modulation of smn2 splicing smn2
HK18106717.9A HK1247103B (zh) 2005-06-23 2018-05-24 調節smn2剪接的組合物和方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
HK18106717.9A HK1247103B (zh) 2005-06-23 2018-05-24 調節smn2剪接的組合物和方法

Country Status (13)

Country Link
US (6) US8361977B2 (de)
EP (5) EP3470072A1 (de)
CY (2) CY2017040I2 (de)
DK (3) DK2548560T3 (de)
ES (3) ES2702531T3 (de)
HK (2) HK1181307A1 (de)
HU (2) HUE027486T2 (de)
LT (2) LT3308788T (de)
LU (1) LUC00046I2 (de)
PL (3) PL2548560T3 (de)
PT (3) PT1910395E (de)
SI (2) SI3308788T1 (de)
WO (1) WO2007002390A2 (de)

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
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
EP3470072A1 (de) 2005-06-23 2019-04-17 Biogen MA Inc. Zusammensetzungen und verfahren zur modulation der smn2-spleissung
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
EP2219680A2 (de) * 2007-11-13 2010-08-25 Isis Pharmaceuticals, Inc. Verbindungen und verfahren zur modulation der proteinexpression
US8633019B2 (en) 2008-05-27 2014-01-21 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
US8986935B2 (en) 2008-08-13 2015-03-24 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
IT1398361B1 (it) * 2009-01-21 2013-02-22 Fond Santa Lucia Uso di mutanti dominanti negativi di sam68 per il trattamento della sma
DK2417257T3 (da) * 2009-04-10 2016-06-06 Ass Inst De Myologie Tricyclo-dna-antisense-oligonukleotider, sammensætninger, og fremgangsmåder til behandlingen af sygdom
US20120149757A1 (en) * 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
CN116440150A (zh) * 2009-06-17 2023-07-18 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法
AU2010270714B2 (en) 2009-07-06 2015-08-13 Wave Life Sciences Ltd. Novel nucleic acid prodrugs and methods 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
US8779128B2 (en) 2010-05-28 2014-07-15 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
EP2582397A4 (de) * 2010-06-15 2014-10-29 Isis Pharmaceuticals Inc Verbindungen und verfahren zur modulierung der interaktion zwischen proteinen und zielnukleinsäuren
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
US10428019B2 (en) 2010-09-24 2019-10-01 Wave Life Sciences Ltd. Chiral auxiliaries
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
DK2638163T3 (en) 2010-11-12 2017-07-24 Massachusetts Gen Hospital 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
EP2704749A1 (de) 2011-05-05 2014-03-12 Sarepta Therapeutics, Inc. Peptid-oligonukleotidkonjugate
AU2012272656A1 (en) * 2011-06-23 2014-02-06 Cold Spring Harbor Laboratory Phenocopy model of disease
DK2734208T3 (en) 2011-07-19 2017-06-19 Wave Life Sciences Ltd PROCEDURES FOR SYNTHESIS OF FUNCTIONALIZED NUCLEIC ACIDS
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
US20140343127A1 (en) 2011-11-11 2014-11-20 Santaris Pharma A/S a corporation Compounds for the modulation of smn2 splicing
JP2015500204A (ja) 2011-11-18 2015-01-05 サレプタ セラピューティクス， インコーポレイテッド 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット
ES2727481T3 (es) * 2011-11-30 2019-10-16 Sarepta Therapeutics Inc Inclusión inducida de exón en atrofia muscular espinal
JP6317675B2 (ja) 2011-11-30 2018-04-25 サレプタ セラピューティクス， インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
AU2012347765B2 (en) * 2011-12-06 2017-05-04 Research Institute At Nationwide Childrens's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
AU2013212758A1 (en) 2012-01-27 2014-08-14 Biomarin Technologies B.V. RNA modulating oligonucleotides with improved characteristics for the treatment of Duchenne and Becker muscular dystrophy
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, 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
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EA201492122A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии utrn
SG11201500232UA (en) 2012-07-13 2015-04-29 Wave Life Sciences Pte Ltd Chiral control
US9598458B2 (en) 2012-07-13 2017-03-21 Wave Life Sciences Japan, Inc. Asymmetric auxiliary group
US9926559B2 (en) 2013-01-09 2018-03-27 Biogen Ma Inc. Compositions and methods for modulation of SMN2 splicing in a subject
EP2946013A1 (de) 2013-01-16 2015-11-25 Iowa State University Research Foundation, Inc. Tiefes intronisches ziel zur spleisskorrektur des spinalen muskelatrophiegens
WO2014172698A1 (en) 2013-04-19 2014-10-23 Isis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
CN105793422B (zh) 2013-09-05 2020-03-03 萨罗塔治疗公司(美国) 酸性α-葡糖苷酶中反义诱导的外显子2纳入
WO2015051283A1 (en) 2013-10-04 2015-04-09 Rana Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
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
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 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
US10160969B2 (en) 2014-01-16 2018-12-25 Wave Life Sciences Ltd. Chiral design
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
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The 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
US10851371B2 (en) 2015-04-10 2020-12-01 Ionis Pharmaceuticals, Inc. Modulation of SMN expression
EP3851531A1 (de) 2015-06-01 2021-07-21 Sarepta Therapeutics, Inc. Antisense-induzierte exonexklusion in typ-vii-kollagen
EP3302489A4 (de) 2015-06-04 2019-02-06 Sarepta Therapeutics, Inc. Verfahren und verbindungen zur behandlung von krankheiten und leiden im zusammenhang mit lymphozyten
CA2996164A1 (en) 2015-08-28 2017-03-09 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
CN108699555A (zh) 2015-10-09 2018-10-23 萨勒普塔医疗公司 用于治疗杜兴肌营养不良和相关病症的组合物和方法
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
CN109312343B (zh) 2015-12-14 2022-09-27 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
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
CA3027288A1 (en) 2016-06-14 2017-12-21 Biogen Ma Inc. Hydrophobic interaction chromatography for purification of oligonucleotides
EP3471779A4 (de) 2016-06-16 2020-07-08 Ionis Pharmaceuticals, Inc. Kombinationen zur modulation der smn-expression
WO2017223258A1 (en) 2016-06-24 2017-12-28 Biogen Ma Inc. Synthesis of thiolated oligonucleotides without a capping step
WO2018009547A1 (en) 2016-07-05 2018-01-11 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Diagnosing col6-related disorders and methods for treating same
AU2017297624B2 (en) * 2016-07-15 2023-11-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of transcript processing
AU2017368050A1 (en) 2016-11-29 2019-06-20 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
HRP20240257T1 (hr) 2017-02-28 2024-05-24 The Trustees Of The University Of Pennsylvania Adeno-povezani virusni (aav) clade f vektor i njegova uporaba
JP2020517638A (ja) 2017-04-20 2020-06-18 エータイアー ファーマ， インコーポレイテッド 肺の炎症を治療するための組成物および方法
WO2019032102A1 (en) 2017-08-09 2019-02-14 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services LINEAR DUPLEX DNA, WITH CLOSED ENDS OF ADENO-ASSOCIATED VIRUSES AND USES THEREOF
RS65031B1 (sr) 2017-08-25 2024-02-29 Stoke Therapeutics Inc Antisens oligomeri za lečenje stanja i bolesti
WO2019075357A1 (en) 2017-10-12 2019-04-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
EP3697910A4 (de) 2017-10-18 2021-07-14 Sarepta Therapeutics, Inc. Antisense-oligomer-verbindungen
SG11202004406VA (en) 2017-11-08 2020-06-29 Avexis Inc Means and method for preparing viral vectors and uses of same
WO2019140231A1 (en) 2018-01-12 2019-07-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
KR20200112906A (ko) 2018-01-25 2020-10-05 바이오젠 엠에이 인코포레이티드 척추 근위축증의 치료 방법
EP3788169A4 (de) * 2018-05-04 2022-08-10 Stoke Therapeutics, Inc. Verfahren und zusammensetzungen zur behandlung von cholesterinesterspeicherkrankheit
JP7478675B2 (ja) 2018-06-08 2024-05-07 ノバルティス アーゲー 薬物製品の効力を測定するための細胞ベースアッセイ
CN113508135A (zh) 2018-10-29 2021-10-15 比奥根Ma公司 增强血脑屏障转运的人源化和稳定化的fc5变体
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
MX2021006359A (es) 2018-11-30 2021-08-11 Novartis Ag Vectores virales de vaa y sus usos.
WO2020191212A1 (en) 2019-03-20 2020-09-24 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
KR20220007104A (ko) 2019-05-08 2022-01-18 바이오젠 엠에이 인코포레이티드 올리고뉴클레오타이드의 수렴형 액상 합성
EP3999643B1 (de) 2019-07-19 2023-11-22 Biogen MA Inc. Verfahren zur behandlung oder vorbeugung von spinaler muskelatrophie
US20220064638A1 (en) * 2020-02-28 2022-03-03 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating smn2
WO2021231107A1 (en) 2020-05-11 2021-11-18 Stoke Therapeutics, Inc. Opa1 antisense oligomers for treatment of conditions and diseases
CA3218805A1 (en) 2021-05-10 2022-11-17 Ziqing QIAN Compositions and methods for intracellular therapeutics
CN118043039A (zh) 2021-10-25 2024-05-14 诺华股份有限公司 用于改善腺相关病毒（aav）递送的方法
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.
NZ266386A (en) 1993-05-11 1997-11-24 Univ North Carolina Use of antisense rna oligonucleotides in regulating gene expression
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
MX9603702A (es) 1994-02-28 1997-12-31 Agritech Technologies Ltd Composiciones de inhibina y métodos de uso en las mismas.
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
AU776362B2 (en) 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
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
CA2437942C (en) 2000-11-09 2013-06-11 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
EP1560931B1 (de) 2002-11-14 2011-07-27 Dharmacon, Inc. Funktionelle und hyperfunktionelle 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
EP3470072A1 (de) 2005-06-23 2019-04-17 Biogen MA Inc. Zusammensetzungen und verfahren zur modulation der smn2-spleissung
US20120021515A1 (en) 2009-02-06 2012-01-26 Swayze Eric E Oligomeric compounds and methods
US20120149757A1 (en) 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
CN116440150A (zh) 2009-06-17 2023-07-18 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法

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