MX391304B - Acido nucleico antisentido. - Google Patents
Acido nucleico antisentido.Info
- Publication number
- MX391304B MX391304B MX2018002955A MX2018002955A MX391304B MX 391304 B MX391304 B MX 391304B MX 2018002955 A MX2018002955 A MX 2018002955A MX 2018002955 A MX2018002955 A MX 2018002955A MX 391304 B MX391304 B MX 391304B
- Authority
- MX
- Mexico
- Prior art keywords
- nucleic acid
- antisense nucleic
- exon
- oligomer
- provides
- Prior art date
Links
- 230000000692 anti-sense effect Effects 0.000 title 1
- 150000007523 nucleic acids Chemical class 0.000 title 1
- 102000039446 nucleic acids Human genes 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 title 1
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- C07H21/04—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 deoxyribosyl 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
- C12N15/113—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
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/313—Phosphorodithioates
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Abstract
La presente invención se refiere a un oligómero que permite la omisión del exón 45 en el gen de la distrofina humana.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2015182145 | 2015-09-15 | ||
| PCT/JP2016/077305 WO2017047707A1 (ja) | 2015-09-15 | 2016-09-15 | アンチセンス核酸 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| MX2018002955A MX2018002955A (es) | 2018-05-02 |
| MX391304B true MX391304B (es) | 2025-03-21 |
Family
ID=58289325
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2018002955A MX391304B (es) | 2015-09-15 | 2016-09-15 | Acido nucleico antisentido. |
Country Status (29)
| Country | Link |
|---|---|
| US (4) | US10144931B2 (es) |
| EP (2) | EP3351633B1 (es) |
| JP (4) | JP6384845B2 (es) |
| KR (3) | KR102473431B1 (es) |
| CN (3) | CN113930426A (es) |
| AU (1) | AU2016324800B2 (es) |
| CA (1) | CA2996280C (es) |
| CO (1) | CO2018002557A2 (es) |
| CY (1) | CY1123119T1 (es) |
| DK (1) | DK3351633T3 (es) |
| ES (1) | ES2808049T3 (es) |
| HR (1) | HRP20201125T1 (es) |
| HU (1) | HUE050061T2 (es) |
| IL (1) | IL258065B (es) |
| LT (1) | LT3351633T (es) |
| MX (1) | MX391304B (es) |
| MY (1) | MY185390A (es) |
| PH (1) | PH12018500568B1 (es) |
| PL (1) | PL3351633T3 (es) |
| PT (1) | PT3351633T (es) |
| RS (1) | RS60493B1 (es) |
| RU (1) | RU2724554C2 (es) |
| SG (1) | SG11201802138TA (es) |
| SI (1) | SI3351633T1 (es) |
| SM (1) | SMT202000379T1 (es) |
| TW (1) | TWI725990B (es) |
| UA (1) | UA123359C2 (es) |
| WO (1) | WO2017047707A1 (es) |
| ZA (1) | ZA201801682B (es) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| UA123359C2 (uk) * | 2015-09-15 | 2021-03-24 | Ніппон Шин'Яку Ко., Лтд. | Антисенсова нуклеїнова кислота |
| MA46427A (fr) | 2015-10-09 | 2018-08-15 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| 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 |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US20210308273A1 (en) | 2018-08-02 | 2021-10-07 | 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 |
| GB201821269D0 (en) * | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| EP4330395A1 (en) | 2021-04-30 | 2024-03-06 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| CN117940564A (zh) * | 2021-06-23 | 2024-04-26 | 日本新药株式会社 | 反义低聚物的组合 |
| 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 |
| 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 |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US20250066396A1 (en) | 2021-12-27 | 2025-02-27 | Nippon Shinyaku Co., Ltd. | Method for producing oligonucleic acid compound |
| EP4486386A1 (en) | 2022-03-03 | 2025-01-08 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
| CA3252177A1 (en) | 2022-03-17 | 2023-09-21 | Sarepta Therapeutics Inc | MORPHOLINO PHOSPHORODIAMIDATE OLIGOMER CONJUGATE |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
Family Cites Families (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US2567664A (en) | 1948-04-22 | 1951-09-11 | Willis G Ewell | Chicken culling device |
| WO1991009033A1 (en) | 1989-12-20 | 1991-06-27 | Anti-Gene Development Group | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| JP2924179B2 (ja) | 1993-02-19 | 1999-07-26 | 日本新薬株式会社 | グリセロール誘導体，デバイス及び医薬組成物 |
| 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 |
| JP4836366B2 (ja) * | 2000-08-25 | 2011-12-14 | 雅文 松尾 | デュシェンヌ型筋ジストロフィー治療剤 |
| 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 |
| EP2386636B1 (en) | 2002-11-25 | 2016-01-27 | Masafumi Matsuo | Ena nucleic acid drugs modifying splicing in mrna precursor |
| CA2524255C (en) * | 2003-03-21 | 2014-02-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| HUE028632T2 (en) | 2004-06-28 | 2016-12-28 | Univ Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP1811024A1 (en) | 2004-10-05 | 2007-07-25 | Nippon Shinyaku Co., Ltd. | Oligo double-stranded rna and medicinal composition |
| WO2006129594A1 (ja) | 2005-05-30 | 2006-12-07 | Nippon Shinyaku Co., Ltd. | 核酸含有複合体製剤の製造方法 |
| EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| WO2008096690A1 (ja) | 2007-02-05 | 2008-08-14 | Nippon Shinyaku Co., Ltd. | ポリエチレングリコール誘導体 |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| EP3238737B1 (en) * | 2007-10-26 | 2022-04-06 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| MX2010004955A (es) | 2007-11-15 | 2010-06-30 | Avi Biopharma Inc | Metodo de sintesis de oligomeros de morfolina. |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| AU2009308167B2 (en) | 2008-10-24 | 2016-03-31 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for DMD |
| EP2344637B2 (en) | 2008-10-27 | 2018-02-28 | BioMarin Technologies B.V. | Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna |
| US20120046342A1 (en) * | 2009-04-24 | 2012-02-23 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
| ITTO20090487A1 (it) * | 2009-06-26 | 2010-12-27 | Univ Ferrara | Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd) |
| US20120270930A1 (en) * | 2009-10-29 | 2012-10-25 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Methods and compositions for dysferlin exon-skipping |
| JP5963678B2 (ja) | 2009-11-12 | 2016-08-03 | ジ ユニバーシティ オブ ウェスタン オーストラリア | 病状を治療するためのアンチセンス分子及び方法 |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| CN103501793A (zh) * | 2011-02-08 | 2014-01-08 | 夏洛特-梅克伦堡医院(商业用名:卡罗来纳保健系统) | 反义寡核苷酸 |
| AU2012360702C1 (en) * | 2011-12-28 | 2018-09-20 | National Center Of Neurology And Psychiatry | Antisense nucleic acid |
| CN104203289B (zh) | 2012-01-27 | 2020-11-03 | 比奥马林技术公司 | 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| EP3608407A1 (en) * | 2012-07-03 | 2020-02-12 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
| KR20150133768A (ko) | 2013-03-15 | 2015-11-30 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 개선된 조성물 |
| UA120849C2 (uk) | 2014-03-12 | 2020-02-25 | Ніппон Шин'Яку Ко., Лтд. | Антисенсовий олігомер |
| JP2015182145A (ja) | 2014-03-20 | 2015-10-22 | キヤノン株式会社 | ロボットシステムの制御方法、およびロボットシステム |
| HRP20200042T1 (hr) | 2014-06-17 | 2020-03-20 | Nippon Shinyaku Co., Ltd. | Suprotne nukleinske kiseline za uporabu u liječenju duchennove mišićne distrofije |
| UA123359C2 (uk) * | 2015-09-15 | 2021-03-24 | Ніппон Шин'Яку Ко., Лтд. | Антисенсова нуклеїнова кислота |
-
2016
- 2016-09-15 UA UAA201802093A patent/UA123359C2/uk unknown
- 2016-09-15 CN CN202111073060.2A patent/CN113930426A/zh active Pending
- 2016-09-15 LT LTEP16846578.9T patent/LT3351633T/lt unknown
- 2016-09-15 US US15/759,267 patent/US10144931B2/en active Active
- 2016-09-15 MY MYPI2018000333A patent/MY185390A/en unknown
- 2016-09-15 AU AU2016324800A patent/AU2016324800B2/en active Active
- 2016-09-15 EP EP16846578.9A patent/EP3351633B1/en active Active
- 2016-09-15 PL PL16846578T patent/PL3351633T3/pl unknown
- 2016-09-15 PT PT168465789T patent/PT3351633T/pt unknown
- 2016-09-15 KR KR1020197010069A patent/KR102473431B1/ko active Active
- 2016-09-15 ES ES16846578T patent/ES2808049T3/es active Active
- 2016-09-15 PH PH1/2018/500568A patent/PH12018500568B1/en unknown
- 2016-09-15 SM SM20200379T patent/SMT202000379T1/it unknown
- 2016-09-15 CN CN201680053726.9A patent/CN108026531B/zh active Active
- 2016-09-15 RS RS20200811A patent/RS60493B1/sr unknown
- 2016-09-15 KR KR1020227012681A patent/KR20220053048A/ko not_active Ceased
- 2016-09-15 SG SG11201802138TA patent/SG11201802138TA/en unknown
- 2016-09-15 WO PCT/JP2016/077305 patent/WO2017047707A1/ja not_active Ceased
- 2016-09-15 RU RU2018113276A patent/RU2724554C2/ru active
- 2016-09-15 CN CN202111073029.9A patent/CN113913426B/zh active Active
- 2016-09-15 SI SI201630848T patent/SI3351633T1/sl unknown
- 2016-09-15 EP EP20181465.4A patent/EP3778895A1/en active Pending
- 2016-09-15 HR HRP20201125TT patent/HRP20201125T1/hr unknown
- 2016-09-15 KR KR1020187001380A patent/KR101968880B1/ko active Active
- 2016-09-15 HU HUE16846578A patent/HUE050061T2/hu unknown
- 2016-09-15 JP JP2017539975A patent/JP6384845B2/ja active Active
- 2016-09-15 DK DK16846578.9T patent/DK3351633T3/da active
- 2016-09-15 CA CA2996280A patent/CA2996280C/en active Active
- 2016-09-15 MX MX2018002955A patent/MX391304B/es unknown
- 2016-09-19 TW TW105130156A patent/TWI725990B/zh active
-
2018
- 2018-03-09 CO CONC2018/0002557A patent/CO2018002557A2/es unknown
- 2018-03-12 ZA ZA2018/01682A patent/ZA201801682B/en unknown
- 2018-03-13 IL IL258065A patent/IL258065B/en unknown
- 2018-07-31 JP JP2018144099A patent/JP6977998B2/ja active Active
- 2018-10-16 US US16/161,946 patent/US10851373B2/en active Active
-
2020
- 2020-07-10 CY CY20201100640T patent/CY1123119T1/el unknown
- 2020-11-17 US US16/950,449 patent/US11981894B2/en active Active
-
2021
- 2021-11-02 JP JP2021179353A patent/JP2022033738A/ja not_active Withdrawn
-
2023
- 2023-12-26 JP JP2023218764A patent/JP2024038104A/ja active Pending
-
2024
- 2024-05-13 US US18/662,046 patent/US20240368597A1/en not_active Abandoned
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| PH12018500568B1 (en) | Antisense nucleic acid | |
| PH12016501761A1 (en) | Antisense nucleic acids | |
| IL271389B1 (en) | Non-viral DNA revenues are targeted | |
| SG11201911430PA (en) | Novel nucleic acid molecules | |
| HUE066467T2 (hu) | Nukleinsav-irányított nukleázok | |
| ZA201706236B (en) | Tat-induced crispr/endonuclease-based gene editing | |
| CO2017000357A2 (es) | Ácidos nucleicos antisentido | |
| WO2016065349A8 (en) | Short non-coding protein regulatory rnas (sprrnas) and methods of use | |
| JOP20200115A1 (ar) | تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات)) | |
| PT3387130T (pt) | Ferramentas de terapia génica para saltar o exão 53 da distrofina | |
| IL276149A (en) | Synthesis of oligonucleotides and nucleic acids | |
| IL257831B2 (en) | Diplosporic garden | |
| IL260122A (en) | Methods and compositions for assembly of nucleic acids | |
| PH12017501864A1 (en) | Compositions and methods for treating autism | |
| IL254304A0 (en) | Nucleoside agents to reduce negative effects of genes involving elongated nucleotide repeats | |
| MX2018005872A (es) | Profarmacos de acido nucleico. | |
| MY209476A (en) | Antisense nucleic acid that induces skipping of exon 50 | |
| EP3631006A4 (en) | DNA STABILIZATION OF RNA | |
| GB201608944D0 (en) | Gene Tharapy | |
| HK40019204B (en) | Nucleic acid products that inhibit the expression of tmprss6 gene and their uses | |
| EA201990115A1 (ru) | Олигомеры для пропуска экзона при мышечной дистрофии | |
| SG10202008046UA (en) | Nucleic acid oligomers and uses therefor | |
| AU2015904479A0 (en) | Compositions and methods for the delivery of agents that inhibit gene expression |