JP2003533986A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2003533986A5 JP2003533986A5 JP2001580347A JP2001580347A JP2003533986A5 JP 2003533986 A5 JP2003533986 A5 JP 2003533986A5 JP 2001580347 A JP2001580347 A JP 2001580347A JP 2001580347 A JP2001580347 A JP 2001580347A JP 2003533986 A5 JP2003533986 A5 JP 2003533986A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- nucleotide sequence
- mrna
- protein
- compound
- Prior art date
- Legal status (The legal status 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 status listed.)
- Pending
Links
- 239000002773 nucleotide Substances 0.000 description 33
- 125000003729 nucleotide group Chemical group 0.000 description 33
- 150000001875 compounds Chemical class 0.000 description 32
- 108020004999 messenger RNA Proteins 0.000 description 20
- 102000004169 proteins and genes Human genes 0.000 description 19
- 108090000623 proteins and genes Proteins 0.000 description 19
- 230000000692 anti-sense effect Effects 0.000 description 14
- 108020005067 RNA Splice Sites Proteins 0.000 description 8
- 210000004027 cell Anatomy 0.000 description 7
- 238000000034 method Methods 0.000 description 6
- KDCGOANMDULRCW-UHFFFAOYSA-N 7H-purine Chemical compound N1=CNC2=NC=NC2=C1 KDCGOANMDULRCW-UHFFFAOYSA-N 0.000 description 4
- IJGRMHOSHXDMSA-UHFFFAOYSA-N Atomic nitrogen Chemical compound N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 description 4
- 108091026890 Coding region Proteins 0.000 description 4
- 101000775732 Homo sapiens Androgen receptor Proteins 0.000 description 4
- 101000928259 Homo sapiens NADPH:adrenodoxin oxidoreductase, mitochondrial Proteins 0.000 description 4
- 102000046818 human AR Human genes 0.000 description 4
- 101001030211 Homo sapiens Myc proto-oncogene protein Proteins 0.000 description 3
- 241000725303 Human immunodeficiency virus Species 0.000 description 3
- 230000000295 complement effect Effects 0.000 description 3
- 210000003527 eukaryotic cell Anatomy 0.000 description 3
- 102000053563 human MYC Human genes 0.000 description 3
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 description 2
- 241000701806 Human papillomavirus Species 0.000 description 2
- 241000702617 Human parvovirus B19 Species 0.000 description 2
- 101710163270 Nuclease Proteins 0.000 description 2
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 description 2
- CZPWVGJYEJSRLH-UHFFFAOYSA-N Pyrimidine Chemical compound C1=CN=CN=C1 CZPWVGJYEJSRLH-UHFFFAOYSA-N 0.000 description 2
- 108091081024 Start codon Proteins 0.000 description 2
- 229910052799 carbon Inorganic materials 0.000 description 2
- 229910052739 hydrogen Inorganic materials 0.000 description 2
- 239000001257 hydrogen Substances 0.000 description 2
- 230000002401 inhibitory effect Effects 0.000 description 2
- 239000000203 mixture Substances 0.000 description 2
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 description 2
- 229910052757 nitrogen Inorganic materials 0.000 description 2
- 229910052698 phosphorus Inorganic materials 0.000 description 2
- 239000011574 phosphorus Substances 0.000 description 2
- 102000040430 polynucleotide Human genes 0.000 description 2
- 108091033319 polynucleotide Proteins 0.000 description 2
- 239000002157 polynucleotide Substances 0.000 description 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 1
- 102000011022 Chorionic Gonadotropin Human genes 0.000 description 1
- 108010062540 Chorionic Gonadotropin Proteins 0.000 description 1
- 108700020121 Human Immunodeficiency Virus-1 rev Proteins 0.000 description 1
- 102000015872 Human beta Subunit Chorionic Gonadotropin Human genes 0.000 description 1
- 108010010590 Human beta Subunit Chorionic Gonadotropin Proteins 0.000 description 1
- YNAVUWVOSKDBBP-UHFFFAOYSA-N Morpholine Chemical group C1COCCN1 YNAVUWVOSKDBBP-UHFFFAOYSA-N 0.000 description 1
- 101710150912 Myc protein Proteins 0.000 description 1
- 230000008878 coupling Effects 0.000 description 1
- 238000010168 coupling process Methods 0.000 description 1
- 238000005859 coupling reaction Methods 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 229940084986 human chorionic gonadotropin Drugs 0.000 description 1
- 230000005764 inhibitory process Effects 0.000 description 1
- 238000007781 pre-processing Methods 0.000 description 1
- 238000002360 preparation method Methods 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US20237600P | 2000-05-04 | 2000-05-04 | |
| US60/202,376 | 2000-05-04 | ||
| PCT/US2001/014410 WO2001083740A2 (en) | 2000-05-04 | 2001-05-04 | Splice-region antisense composition and method |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011131668A Division JP2011172597A (ja) | 2000-05-04 | 2011-06-13 | スプライス領域アンチセンス組成物および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2003533986A JP2003533986A (ja) | 2003-11-18 |
| JP2003533986A5 true JP2003533986A5 (https=) | 2011-07-28 |
Family
ID=22749613
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2001580347A Pending JP2003533986A (ja) | 2000-05-04 | 2001-05-04 | スプライス領域アンチセンス組成物および方法 |
| JP2011131668A Pending JP2011172597A (ja) | 2000-05-04 | 2011-06-13 | スプライス領域アンチセンス組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011131668A Pending JP2011172597A (ja) | 2000-05-04 | 2011-06-13 | スプライス領域アンチセンス組成物および方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (8) | US6784291B2 (https=) |
| EP (1) | EP1282699B1 (https=) |
| JP (2) | JP2003533986A (https=) |
| KR (1) | KR20020097241A (https=) |
| AU (2) | AU5752601A (https=) |
| CA (1) | CA2408011A1 (https=) |
| WO (1) | WO2001083740A2 (https=) |
Families Citing this family (128)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6784291B2 (en) | 2000-05-04 | 2004-08-31 | Avi Biopharma, Inc. | Splice-region antisense composition and method |
| NZ530101A (en) * | 2001-05-17 | 2007-01-26 | Avi Biopharma Inc | Methods for treating cancer by administering an oligomer antisense to c-myc together with a cancer chemotherapeutic agent |
| WO2003070896A2 (en) * | 2002-02-20 | 2003-08-28 | Sirna Therapeutics, Inc | RNA INTERFERENCE MEDIATED INHIBITION OF PROLIFERATING CELL NUCLEAR ANTIGEN (PCNA) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| US9994853B2 (en) | 2001-05-18 | 2018-06-12 | Sirna Therapeutics, Inc. | Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference |
| US20050159381A1 (en) * | 2001-05-18 | 2005-07-21 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of chromosome translocation gene expression using short interfering nucleic acid (siNA) |
| US9181551B2 (en) | 2002-02-20 | 2015-11-10 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| US20090093439A1 (en) * | 2002-02-20 | 2009-04-09 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| US9657294B2 (en) | 2002-02-20 | 2017-05-23 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| AU2003219833A1 (en) * | 2002-02-20 | 2003-09-09 | Sirna Therapeutics Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| US20040224986A1 (en) * | 2002-08-16 | 2004-11-11 | Bart De Corte | Piperidinyl targeting compounds that selectively bind integrins |
| US9034337B2 (en) | 2003-10-31 | 2015-05-19 | Prothena Biosciences Limited | Treatment and delay of outset of synucleinopathic and amyloidogenic disease |
| US8506959B2 (en) * | 2002-11-01 | 2013-08-13 | Neotope Biosciences Limited | Prevention and treatment of synucleinopathic and amyloidogenic disease |
| US20080014194A1 (en) * | 2003-10-31 | 2008-01-17 | Elan Pharmaceuticals, Inc. | Prevention and Treatment of Synucleinopathic and Amyloidogenic Disease |
| TW200509968A (en) * | 2002-11-01 | 2005-03-16 | Elan Pharm Inc | Prevention and treatment of synucleinopathic disease |
| US7358331B2 (en) | 2003-05-19 | 2008-04-15 | Elan Pharmaceuticals, Inc. | Truncated fragments of alpha-synuclein in Lewy body disease |
| US20080311556A1 (en) * | 2003-08-07 | 2008-12-18 | Iversen Patrick L | Sense Antiviral Compound and Method for Treating Ssrna Viral Infection |
| WO2005027833A2 (en) * | 2003-09-12 | 2005-03-31 | Avi Biopharma, Inc. | Compound and method for treating androgen-independent prostate cancer |
| US20050222068A1 (en) * | 2003-10-23 | 2005-10-06 | Mourich Dan V | Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells |
| AR041407A1 (es) * | 2003-09-26 | 2005-05-18 | Nestor Alberto Kerner | Oligonucleotidos antiandrogenos utilizables en el tratamiento de patologias dermatologicas relacionadas con el metabolismo de los androgenos, sus composiciones farmaceuticas y sus usos y metodos de tratamiento |
| US20050113328A1 (en) * | 2003-11-06 | 2005-05-26 | Devi Gayathri R. | Method and antisense compound for potentiating anti-cancer agents |
| WO2005047860A2 (en) | 2003-11-08 | 2005-05-26 | Elan Pharmaceuticals, Inc. | Antibodies to alpha-synuclein |
| US20050171044A1 (en) * | 2003-12-24 | 2005-08-04 | Stein David A. | Oligonucleotide compound and method for treating nidovirus infections |
| CA2553104A1 (en) | 2004-01-23 | 2005-08-11 | Avi Biopharma, Inc. | Antisense oligomers and methods for inducing immune tolerance and immunosuppression |
| US7402574B2 (en) * | 2004-03-12 | 2008-07-22 | Avi Biopharma, Inc. | Antisense composition and method for treating cancer |
| US10508277B2 (en) | 2004-05-24 | 2019-12-17 | Sirna Therapeutics, Inc. | Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference |
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP2351839A3 (en) * | 2004-07-02 | 2011-10-05 | AVI BioPharma, Inc. | Antisense antibacterial method and compound |
| EA013752B1 (ru) * | 2004-08-09 | 2010-06-30 | Элан Фармасьютикалз, Инк. | Предупреждение и лечение синуклеинопатических и амилоидогенных заболеваний |
| US8129352B2 (en) * | 2004-09-16 | 2012-03-06 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating ssRNA viral infection |
| US8357664B2 (en) * | 2004-10-26 | 2013-01-22 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating influenza viral infection |
| US7524829B2 (en) * | 2004-11-01 | 2009-04-28 | Avi Biopharma, Inc. | Antisense antiviral compounds and methods for treating a filovirus infection |
| EP1855694B1 (en) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition for treating muscle atrophy |
| US20060240032A1 (en) * | 2005-03-31 | 2006-10-26 | Hinrichs David J | Immunomodulating compositions and methods for use in the treatment of human autoimmune diseases |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| US8524676B2 (en) * | 2005-09-08 | 2013-09-03 | Sarepta Therapeutics, Inc. | Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds |
| WO2007030576A2 (en) * | 2005-09-08 | 2007-03-15 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating picornavirus infection |
| WO2007035962A2 (en) * | 2005-09-23 | 2007-03-29 | California Institute Of Technology | Gene blocking method |
| US8501704B2 (en) | 2005-11-08 | 2013-08-06 | Sarepta Therapeutics, Inc. | Immunosuppression compound and treatment method |
| US7825099B2 (en) * | 2006-01-20 | 2010-11-02 | Quark Pharmaceuticals, Inc. | Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes |
| US7582615B2 (en) * | 2006-03-07 | 2009-09-01 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating arenavirus infection |
| PL2735568T3 (pl) | 2006-05-10 | 2018-02-28 | Sarepta Therapeutics, Inc. | Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami |
| US8785407B2 (en) * | 2006-05-10 | 2014-07-22 | Sarepta Therapeutics, Inc. | Antisense antiviral agent and method for treating ssRNA viral infection |
| US20070265215A1 (en) * | 2006-05-11 | 2007-11-15 | Iversen Patrick L | Antisense restenosis composition and method |
| US8147833B2 (en) * | 2007-02-23 | 2012-04-03 | Neotope Biosciences Limited | Prevention and treatment of synucleinopathic and amyloidogenic disease |
| PL2583978T3 (pl) * | 2007-02-23 | 2016-07-29 | Prothena Biosciences Ltd Co | Profilaktyka i leczenie chorób synukleinopatycznych i amyloidogennych |
| US8450290B2 (en) | 2007-11-26 | 2013-05-28 | Enzon Pharmaceuticals, Inc. | Methods for treating androgen receptor dependent disorders including cancers |
| WO2009068033A2 (en) | 2007-11-26 | 2009-06-04 | Santaris Pharma A/S | Lna antagonists targeting the androgen receptor |
| EP2235034B1 (en) | 2007-12-28 | 2016-11-23 | Sarepta Therapeutics, Inc. | Immunomodulatory agents and methods of use |
| BR122020021379B1 (pt) | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| EP2376633A1 (en) * | 2008-12-17 | 2011-10-19 | AVI BioPharma, Inc. | Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis |
| US20120156138A1 (en) * | 2009-04-14 | 2012-06-21 | Smith Larry J | Methods and Compositions for the Treatment of Medical Conditions Involving Cellular Reprogramming |
| AU2010306940A1 (en) | 2009-10-12 | 2012-06-07 | Smith, Larry | Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro |
| TR201816523T4 (tr) | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| EP2499248B1 (en) | 2009-11-13 | 2017-01-04 | Sarepta Therapeutics, Inc. | Antisense antiviral compound and method for treating influenza viral infection |
| US9050373B2 (en) | 2010-05-13 | 2015-06-09 | The Charlotte-Mecklenburg Hospital Authority | Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same |
| KR20200133284A (ko) | 2010-05-28 | 2020-11-26 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| US8198429B2 (en) | 2010-08-09 | 2012-06-12 | Avi Biopharma, Inc. | Antisense antiviral compounds and methods for treating a filovirus infection |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US10017763B2 (en) | 2010-09-03 | 2018-07-10 | Sarepta Therapeutics, Inc. | dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups |
| WO2012058210A1 (en) | 2010-10-29 | 2012-05-03 | Merck Sharp & Dohme Corp. | RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA) |
| CA2817256A1 (en) | 2010-11-12 | 2012-05-18 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| AU2011326034B2 (en) | 2010-11-12 | 2016-02-04 | Roche Innovation Center Copenhagen A/S | Compositions and methods for treating androgen receptor dependent disorders including cancers |
| EP2672977A1 (en) | 2011-02-08 | 2013-12-18 | The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System | Antisense oligonucleotides |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| EP2725103A3 (en) | 2011-11-14 | 2016-01-06 | Silenseed Ltd | Methods and compositions for treating prostate cancer |
| WO2013074814A2 (en) * | 2011-11-15 | 2013-05-23 | University Of Utah Research Fourdation | Morpholinos, morpholino upregulating, and associated methods |
| HK1201514A1 (en) | 2011-11-18 | 2015-09-04 | 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 |
| JP6496549B2 (ja) | 2011-11-30 | 2019-04-03 | サレプタ セラピューティクス， インコーポレイテッド | 脊髄性筋萎縮症における誘発されたエクソン包含 |
| WO2013159051A1 (en) | 2012-04-19 | 2013-10-24 | University Of Utah Research Foundation | Morpholino-mediated increase in soluble flt-1 expression results in decreased ocular and tumor neovascularization |
| EA201492116A1 (ru) | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
| SG11201407483YA (en) | 2012-05-16 | 2014-12-30 | Rana Therapeutics Inc | Compositions and methods for modulating smn gene family expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| AR092982A1 (es) | 2012-10-11 | 2015-05-13 | Isis Pharmaceuticals Inc | Modulacion de la expresion de receptores androgenicos |
| CA3201145A1 (en) | 2012-10-26 | 2014-05-01 | Geron Corporation | C-myc antisense oligonucleotides and methods for using the same to treat cell-proliferative disorders |
| US9228189B2 (en) * | 2012-10-26 | 2016-01-05 | Geron Corporation | C-myc antisense oligonucleotides and methods for using the same to treat cell-proliferative disorders |
| EP3696276A1 (en) | 2013-02-25 | 2020-08-19 | Novartis AG | Novel androgen receptor mutation |
| CN110218727A (zh) | 2013-03-14 | 2019-09-10 | 萨勒普塔医疗公司 | 用于治疗肌营养不良的外显子跳跃组合物 |
| US20140329762A1 (en) | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| EP2983676B1 (en) * | 2013-04-12 | 2019-03-20 | The Curators of the University of Missouri | Smn2 element 1 antisense compositions and methods and uses thereof |
| JP2016531570A (ja) | 2013-08-16 | 2016-10-13 | ラナ セラピューティクス インコーポレイテッド | ユークロマチン領域を標的とするオリゴヌクレオチド |
| US11236338B2 (en) * | 2013-09-05 | 2022-02-01 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| WO2015051283A1 (en) | 2013-10-04 | 2015-04-09 | Rana Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| 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 |
| 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 |
| US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
| US10905709B2 (en) | 2015-08-28 | 2021-02-02 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| IL295755A (en) | 2015-10-09 | 2022-10-01 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| 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 |
| WO2017079570A2 (en) * | 2015-11-04 | 2017-05-11 | Duke University | Splise-switching oligonucleotides and methods of use |
| 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 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| JP2019513371A (ja) | 2016-04-01 | 2019-05-30 | アビディティー バイオサイエンシーズ エルエルシー | 核酸ポリペプチド組成物とその使用 |
| WO2017184529A1 (en) | 2016-04-18 | 2017-10-26 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| BR112018074346B1 (pt) | 2016-05-24 | 2023-01-03 | Sarepta Therapeutics, Inc. | Composto oligomérico e processo para preparar um composto oligomérico |
| US10947533B2 (en) | 2016-05-24 | 2021-03-16 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MD3464306T2 (ro) | 2016-05-24 | 2024-08-31 | Sarepta Therapeutics Inc | Procedee de preparare a oligomerilor morfolino fosforodiamidați |
| CA3024456A1 (en) | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MX2019008199A (es) | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| KR102318434B1 (ko) | 2017-08-25 | 2021-11-01 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| AU2018378812B2 (en) | 2017-12-06 | 2025-05-22 | Avidity Biosciences, Inc. | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
| EP3765030A4 (en) * | 2018-03-16 | 2022-01-26 | Sarepta Therapeutics, Inc. | CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION |
| CA3099280A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| TW202449155A (zh) * | 2018-07-27 | 2024-12-16 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| AU2020227825B2 (en) | 2019-02-27 | 2026-03-26 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| AU2019442723A1 (en) * | 2019-04-25 | 2021-09-23 | Electrolux Appliances Aktiebolag | Dishwasher |
| WO2020219820A1 (en) | 2019-04-25 | 2020-10-29 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| WO2021188390A1 (en) | 2020-03-19 | 2021-09-23 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
| CN115867657A (zh) | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| JP2024525608A (ja) | 2021-07-09 | 2024-07-12 | ダイン セラピューティクス，インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体および製剤 |
| EP4458833A4 (en) | 2021-12-27 | 2025-12-31 | Nippon Shinyaku Co Ltd | PROCESS FOR THE PRODUCTION OF OLIGONUCLEIC ACID COMPOUNDS |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
| US20250145992A1 (en) * | 2023-10-18 | 2025-05-08 | University Of Massachusetts | Oligonucleotides with ethylene glycol modification |
Family Cites Families (88)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1986005519A1 (en) * | 1985-03-15 | 1986-09-25 | James Summerton | Polynucleotide assay reagent and method |
| US5034506A (en) * | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5521063A (en) * | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5506337A (en) * | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5185444A (en) * | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5217866A (en) * | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5166315A (en) * | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5948634A (en) * | 1988-12-21 | 1999-09-07 | The General Hospital Coporation | Neural thread protein gene expression and detection of alzheimer's disease |
| EP0421696A3 (en) * | 1989-10-02 | 1992-01-29 | Motorola Inc. | Staggered access memory |
| US5914396A (en) * | 1990-01-11 | 1999-06-22 | Isis Pharmaceuticals, Inc. | 2'-O-modified nucleosides and phosphoramidites |
| US5852188A (en) * | 1990-01-11 | 1998-12-22 | Isis Pharmaceuticals, Inc. | Oligonucleotides having chiral phosphorus linkages |
| US5140592A (en) * | 1990-03-02 | 1992-08-18 | Sf2 Corporation | Disk array system |
| AU659482B2 (en) | 1991-06-28 | 1995-05-18 | Massachusetts Institute Of Technology | Localized oligonucleotide therapy |
| US5858684A (en) * | 1991-08-23 | 1999-01-12 | The Brigham And Women's Hospital, Inc. | Method of screening calcium receptor-active molecules |
| US5526507A (en) * | 1992-01-06 | 1996-06-11 | Hill; Andrew J. W. | Computer memory array control for accessing different memory banks simullaneously |
| AU670125B2 (en) | 1992-09-15 | 1996-07-04 | Immunex Corporation | Method of treating tnf-dependent inflammation using tumor necrosis factor antagonists |
| DE69435005T2 (de) * | 1993-05-11 | 2008-04-17 | The University Of North Carolina At Chapel Hill | Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung |
| KR960703170A (ko) | 1993-06-23 | 1996-06-19 | 알버트 디. 프리센. | 안티센스 올리고뉴클레오티드 및 인간면역결핍바이러스감염에서 그것의 치료적이용(antisense oligonucleotides and therapeutic use thereof in human immunodeficiency virus infection) |
| US5548711A (en) * | 1993-08-26 | 1996-08-20 | Emc Corporation | Method and apparatus for fault tolerant fast writes through buffer dumping |
| US6133242A (en) * | 1993-10-15 | 2000-10-17 | Thomas Jefferson Univerisity | Inhibition of extracellular matrix synthesis by antisense compounds directed to nuclear proto-oncogenes |
| US6060456A (en) * | 1993-11-16 | 2000-05-09 | Genta Incorporated | Chimeric oligonucleoside compounds |
| GB9510718D0 (en) * | 1995-05-26 | 1995-07-19 | Sod Conseils Rech Applic | Antisense oligonucleotides |
| US5632013A (en) * | 1995-06-07 | 1997-05-20 | International Business Machines Corporation | Memory and system for recovery/restoration of data using a memory controller |
| US7803782B2 (en) * | 2003-05-28 | 2010-09-28 | Roche Madison Inc. | Intravenous delivery of polynucleotides to cells in mammalian limb |
| US5790775A (en) * | 1995-10-23 | 1998-08-04 | Digital Equipment Corporation | Host transparent storage controller failover/failback of SCSI targets and associated units |
| AU2585197A (en) | 1996-03-20 | 1997-10-10 | Regents Of The University Of California, The | Antisense approach to gene inhibition |
| US5840579A (en) * | 1996-05-01 | 1998-11-24 | The Johns Hopkins University | Nucleic acids encoding p53 mutations which suppress p53 cancer mutations |
| US5933653A (en) * | 1996-05-31 | 1999-08-03 | Emc Corporation | Method and apparatus for mirroring data in a remote data storage system |
| US5776905A (en) * | 1996-08-08 | 1998-07-07 | The Board Of Trustees Of The Leland Stamford Junior University | Apoptotic regression of intimal vascular lesions |
| US6073218A (en) * | 1996-12-23 | 2000-06-06 | Lsi Logic Corp. | Methods and apparatus for coordinating shared multiple raid controller access to common storage devices |
| US6073209A (en) * | 1997-03-31 | 2000-06-06 | Ark Research Corporation | Data storage controller providing multiple hosts with access to multiple storage subsystems |
| CN1263473A (zh) * | 1997-05-21 | 2000-08-16 | 利兰·斯坦福青年大学托管委员会 | 增加跨生物膜转运的组合物和方法 |
| US6061772A (en) * | 1997-06-30 | 2000-05-09 | Sun Microsystems, Inc. | Split write data processing mechanism for memory controllers utilizing inactive periods during write data processing for other transactions |
| US6103466A (en) * | 1997-07-14 | 2000-08-15 | University Of Liege | Double-muscling in mammals |
| US6133246A (en) * | 1997-08-13 | 2000-10-17 | Isis Pharmaceuticals Inc. | Antisense oligonucleotide compositions and methods for the modulation of JNK proteins |
| CA2248762A1 (en) | 1997-10-22 | 1999-04-22 | University Technologies International, Inc. | Antisense oligodeoxynucleotides regulating expression of tnf-.alpha. |
| US6228579B1 (en) * | 1997-11-14 | 2001-05-08 | San Diego State University Foundation | Method for identifying microbial proliferation genes |
| US6085333A (en) * | 1997-12-19 | 2000-07-04 | Lsi Logic Corporation | Method and apparatus for synchronization of code in redundant controllers in a swappable environment |
| US6041381A (en) * | 1998-02-05 | 2000-03-21 | Crossroads Systems, Inc. | Fibre channel to SCSI addressing method and system |
| US6021454A (en) * | 1998-03-27 | 2000-02-01 | Adaptec, Inc. | Data transfer between small computer system interface systems |
| US6020135A (en) * | 1998-03-27 | 2000-02-01 | Affymetrix, Inc. | P53-regulated genes |
| US6172216B1 (en) * | 1998-10-07 | 2001-01-09 | Isis Pharmaceuticals Inc. | Antisense modulation of BCL-X expression |
| US6210892B1 (en) * | 1998-10-07 | 2001-04-03 | Isis Pharmaceuticals, Inc. | Alteration of cellular behavior by antisense modulation of mRNA processing |
| US6214986B1 (en) | 1998-10-07 | 2001-04-10 | Isis Pharmaceuticals, Inc. | Antisense modulation of bcl-x expression |
| US20030064944A1 (en) * | 2001-06-21 | 2003-04-03 | Isis Pharmaceuticals Inc. | Antisense modulation of transforming growth factor beta receptor II expression |
| KR20010102992A (ko) * | 1999-01-29 | 2001-11-17 | 추후보정 | 표적 ｒｎａ를 검출하는 비-침입적 방법 |
| US7094765B1 (en) * | 1999-01-29 | 2006-08-22 | Avi Biopharma, Inc. | Antisense restenosis composition and method |
| EP1159289A1 (en) | 1999-03-11 | 2001-12-05 | Gryphon Sciences | Chemical synthesis and use of soluble membrane protein receptor domains |
| US6136603A (en) | 1999-03-26 | 2000-10-24 | Isis Pharmaceuticals Inc. | Antisense modulation of interleukin-5 signal transduction |
| US20020049173A1 (en) * | 1999-03-26 | 2002-04-25 | Bennett C. Frank | Alteration of cellular behavior by antisense modulation of mRNA processing |
| US5998148A (en) * | 1999-04-08 | 1999-12-07 | Isis Pharmaceuticals Inc. | Antisense modulation of microtubule-associated protein 4 expression |
| JP2000325085A (ja) | 1999-05-21 | 2000-11-28 | Masafumi Matsuo | デュシェンヌ型筋ジストロフィー治療剤 |
| US6043091A (en) * | 1999-07-19 | 2000-03-28 | Isis Pharmaceuticals Inc. | Antisense modulation of liver glycogen phosphorylase expression |
| JP2003516151A (ja) * | 1999-11-29 | 2003-05-13 | エイブイアイ バイオファーマ， インコーポレイテッド | 細菌１６Ｓおよび２３ＳｒＲＮＡに標的化された、荷電していないアンチセンスオリゴヌクレオチド、ならびにその使用 |
| AU781676B2 (en) * | 2000-01-04 | 2005-06-02 | Avi Biopharma, Inc. | Antisense antibacterial cell division composition and method |
| JP2004509604A (ja) | 2000-03-28 | 2004-04-02 | アイシス・ファーマシューティカルス・インコーポレーテッド | ｍＲＮＡプロセッシングのアンチセンスモジュレーションによる、細胞行動の改変 |
| US6653467B1 (en) * | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
| US6784291B2 (en) | 2000-05-04 | 2004-08-31 | Avi Biopharma, Inc. | Splice-region antisense composition and method |
| EP1301525B1 (en) * | 2000-07-06 | 2015-09-02 | Sarepta Therapeutics, Inc. | Transforming growth factor beta (tgf-beta) blocking agent-treated stem cell composition and method |
| WO2002008893A1 (en) | 2000-07-21 | 2002-01-31 | Antevista Gmbh | A microprocessor having an instruction format containing explicit timing information |
| US6727355B2 (en) * | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| US20040091897A1 (en) | 2001-03-19 | 2004-05-13 | John Todd | Disease associated polymorphism in the ctla-4 locus |
| US20030224353A1 (en) * | 2001-10-16 | 2003-12-04 | Stein David A. | Antisense antiviral agent and method for treating ssRNA viral infection |
| JP2005507660A (ja) * | 2001-10-16 | 2005-03-24 | アビ バイオファーマ， インコーポレイテッド | ｓｓＲＮＡウイルス感染の処置のためのアンチセンス抗ウイルス剤および方法 |
| EP1478730A4 (en) | 2002-02-20 | 2006-01-25 | Sirna Therapeutics Inc | INTERFERENCE RNA-INDUCED INHIBITION OF THE GENE EXPRESSION OF SUPERFAMILY TFN AND TFN RECEPTOR SUPERFAMILY USING SHORT INTERFERENCE NUCLEIC ACID (SINA) |
| DE10228103A1 (de) | 2002-06-24 | 2004-01-15 | Bayer Cropscience Ag | Fungizide Wirkstoffkombinationen |
| EP2392660A3 (en) | 2002-11-25 | 2012-03-28 | Masafumi Matsuo | ENA Nucleic Acid Drugs Modifying Splicing in mRNA Precursor |
| AU2003225410A1 (en) | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| US7049765B1 (en) * | 2003-04-11 | 2006-05-23 | Tremaine Sr John M | Transformer for dimmer switch or on/off switch and method of use |
| US20050202531A1 (en) * | 2003-11-03 | 2005-09-15 | Compugen Ltd. | CD40 splice variants, compositions for making and methods of using the same |
| AU2005218759B2 (en) * | 2004-02-27 | 2009-07-16 | Antisense Pharma Gmbh | Pharmaceutical composition |
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| RU2346996C2 (ru) | 2004-06-29 | 2009-02-20 | ЮРОПИЭН НИКЕЛЬ ПиЭлСи | Усовершенствованное выщелачивание основных металлов |
| US8357664B2 (en) | 2004-10-26 | 2013-01-22 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating influenza viral infection |
| EP1855694B1 (en) * | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition for treating muscle atrophy |
| JP2008535868A (ja) | 2005-04-14 | 2008-09-04 | ザ ユニバーシティー オブ クイーンズランド | 免疫調節組成物およびそのための使用 |
| US7930013B2 (en) | 2005-06-29 | 2011-04-19 | Compumedics Limited | Sensor assembly with conductive bridge |
| US8501704B2 (en) * | 2005-11-08 | 2013-08-06 | Sarepta Therapeutics, Inc. | Immunosuppression compound and treatment method |
| US20070111962A1 (en) * | 2005-11-08 | 2007-05-17 | Mourich Dan V | Immunosuppression compound and treatment method |
| WO2007058894A2 (en) | 2005-11-10 | 2007-05-24 | The University Of North Carolina At Chapel Hill | Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease |
| US7785834B2 (en) * | 2005-11-10 | 2010-08-31 | Ercole Biotech, Inc. | Soluble TNF receptors and their use in treatment of disease |
| WO2008051306A1 (en) | 2006-10-20 | 2008-05-02 | Ercole Biotech, Inc. | Soluble tnf receptors and their use in treatment of disease |
| PL2735568T3 (pl) | 2006-05-10 | 2018-02-28 | Sarepta Therapeutics, Inc. | Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami |
| TW200801513A (en) | 2006-06-29 | 2008-01-01 | Fermiscan Australia Pty Ltd | Improved process |
| US20120142759A1 (en) | 2006-11-10 | 2012-06-07 | Sazani Peter L | Soluble thf receptors and their use in treatment of disease |
| CA2689602A1 (en) * | 2007-06-06 | 2008-12-18 | Avi Biopharma, Inc. | Soluble her2 and her3 splice variant proteins, splice-switching oligonucleotides, and their use in the treatment of disease |
| EP2235034B1 (en) * | 2007-12-28 | 2016-11-23 | Sarepta Therapeutics, Inc. | Immunomodulatory agents and methods of use |
-
2001
- 2001-05-04 US US09/848,868 patent/US6784291B2/en not_active Expired - Lifetime
- 2001-05-04 JP JP2001580347A patent/JP2003533986A/ja active Pending
- 2001-05-04 AU AU5752601A patent/AU5752601A/xx active Pending
- 2001-05-04 CA CA002408011A patent/CA2408011A1/en not_active Abandoned
- 2001-05-04 EP EP01931051A patent/EP1282699B1/en not_active Expired - Lifetime
- 2001-05-04 AU AU2001257526A patent/AU2001257526B2/en not_active Expired
- 2001-05-04 KR KR1020027014612A patent/KR20020097241A/ko not_active Ceased
- 2001-05-04 WO PCT/US2001/014410 patent/WO2001083740A2/en not_active Ceased
-
2004
- 2004-07-16 US US10/893,086 patent/US20040266720A1/en not_active Abandoned
-
2006
- 2006-05-11 US US11/433,214 patent/US8067569B2/en not_active Expired - Fee Related
-
2011
- 2011-06-13 JP JP2011131668A patent/JP2011172597A/ja active Pending
- 2011-08-26 US US13/219,409 patent/US8436163B2/en not_active Expired - Fee Related
-
2013
- 2013-04-09 US US13/859,518 patent/US8895722B2/en not_active Expired - Fee Related
-
2014
- 2014-11-06 US US14/535,098 patent/US9416361B2/en not_active Expired - Fee Related
-
2016
- 2016-07-08 US US15/205,722 patent/US20170159050A1/en not_active Abandoned
-
2017
- 2017-10-03 US US15/723,966 patent/US10533174B2/en not_active Expired - Fee Related
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2003533986A5 (https=) | ||
| JP7116212B2 (ja) | Ｌｐａの遺伝子発現の阻害のための組成物及び方法 | |
| TWI823866B (zh) | 用於抑制類血管生成素蛋白-3(ANGPTL3)表現的RNAi藥劑及組合物及使用方法 | |
| TWI840321B (zh) | 用於B型肝炎病毒感染之RNAi藥劑 | |
| US10246707B2 (en) | Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means | |
| JP2023076634A (ja) | α－１アンチトリプシン（ＡＡＴ）ＲＮＡｉ剤、ＡＡＴ ＲＮＡｉ剤を含む組成物、及び使用方法。 | |
| US8802645B2 (en) | Molecule for treating an inflammatory disorder | |
| TW202415389A (zh) | 用於抑制脂蛋白元C-III (APOC3)表現之RNAi試劑及組合物 | |
| JP2011172597A (ja) | スプライス領域アンチセンス組成物および方法 | |
| EP4389893A1 (en) | Human transferrin receptor binding peptide-drug conjugate | |
| JP2011505833A5 (https=) | ||
| EA035756B1 (ru) | Композиции и способы ингибирования экспрессии генов вируса гепатита в | |
| CN102459302A (zh) | 核酸递送组合物及其使用方法 | |
| CN106535917A (zh) | 用于抑制α‑1抗胰蛋白酶基因表达的组合物及方法 | |
| JP2023531511A5 (https=) | ||
| JP2001512666A5 (https=) | ||
| JP2023503635A (ja) | Ｒｎａ分子を合成する方法 | |
| WO2022248879A1 (en) | Composition and method for adar-mediated rna editing | |
| CN114555188A (zh) | 治疗apoc3相关疾病和病症的方法 | |
| CN119137272A (zh) | 血管紧张素原调节组合物和其使用方法 | |
| EP4381065A2 (en) | Method | |
| JP2004526737A5 (https=) | ||
| Tomita et al. | Antisense oligonucleotides as a powerful molecular strategy for gene therapy in cardiovascular diseases | |
| TW202020153A (zh) | 用於肌肉萎縮症之外顯子跳躍寡聚物 | |
| US20220251556A1 (en) | Enhanced oligonucleotides for inhibiting rtel1 expression |