NO20084488L - Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewith - Google Patents
Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewithInfo
- Publication number
- NO20084488L NO20084488L NO20084488A NO20084488A NO20084488L NO 20084488 L NO20084488 L NO 20084488L NO 20084488 A NO20084488 A NO 20084488A NO 20084488 A NO20084488 A NO 20084488A NO 20084488 L NO20084488 L NO 20084488L
- Authority
- NO
- Norway
- Prior art keywords
- treatment
- functional protein
- methods
- dna
- production
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title abstract 5
- 108020004485 Nonsense Codon Proteins 0.000 title abstract 4
- 230000037434 nonsense mutation Effects 0.000 title abstract 4
- 102000004169 proteins and genes Human genes 0.000 title abstract 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 208000035475 disorder Diseases 0.000 title 1
- 150000007523 nucleic acids Chemical group 0.000 abstract 2
- 201000010099 disease Diseases 0.000 abstract 1
- 230000002265 prevention Effects 0.000 abstract 1
Classifications
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
- A61K31/4245—Oxadiazoles
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- A—HUMAN NECESSITIES
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- A—HUMAN NECESSITIES
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- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
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- A—HUMAN NECESSITIES
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- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
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- A—HUMAN NECESSITIES
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- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
- A61K31/415—1,2-Diazoles
- A61K31/4162—1,2-Diazoles condensed with heterocyclic ring systems
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- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
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- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/46—8-Azabicyclo [3.2.1] octane; Derivatives thereof, e.g. atropine, cocaine
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Landscapes
- Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Diabetes (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Hematology (AREA)
- Endocrinology (AREA)
- Emergency Medicine (AREA)
- Obesity (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pulmonology (AREA)
- Urology & Nephrology (AREA)
- Ophthalmology & Optometry (AREA)
- Toxicology (AREA)
- Psychology (AREA)
- Psychiatry (AREA)
- Otolaryngology (AREA)
- Hospice & Palliative Care (AREA)
- Vascular Medicine (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Child & Adolescent Psychology (AREA)
- Dermatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Foreliggende oppfinnelse angår det funksjonelle proteinet kodet av nukleinsyresekvenser som innbefatter en ikke-sensmutasjon. Foreliggende oppfinnelse angår også fremgangsmåter for produksjon av funksjonelle proteinet kodet av nukleinsyresekvenser som innbefatter en ikke-sensmutasjon og anvendelsen av slike proteinet for hindring, håndtering og/eller behandling av sykdommer assosiert med ikke-sensmutasjoner i et gen.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US78733306P | 2006-03-30 | 2006-03-30 | |
US81308506P | 2006-06-12 | 2006-06-12 | |
PCT/US2007/008268 WO2007117438A2 (en) | 2006-03-30 | 2007-03-29 | Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith |
Publications (1)
Publication Number | Publication Date |
---|---|
NO20084488L true NO20084488L (no) | 2008-12-19 |
Family
ID=38480572
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
NO20084488A NO20084488L (no) | 2006-03-30 | 2008-10-24 | Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewith |
Country Status (15)
Country | Link |
---|---|
US (2) | US9289398B2 (no) |
EP (2) | EP2007365A2 (no) |
JP (2) | JP5367563B2 (no) |
CN (1) | CN101505739A (no) |
AU (1) | AU2007235524B2 (no) |
BR (1) | BRPI0710213A2 (no) |
CA (1) | CA2647903C (no) |
IL (2) | IL194454A (no) |
IN (1) | IN2014CN04406A (no) |
MX (1) | MX2008012515A (no) |
NO (1) | NO20084488L (no) |
NZ (2) | NZ571899A (no) |
RU (1) | RU2462246C2 (no) |
SG (1) | SG170819A1 (no) |
WO (1) | WO2007117438A2 (no) |
Families Citing this family (18)
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EA009120B1 (ru) | 2003-04-11 | 2007-10-26 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Соединения 1,2,4-оксадиазолбензойной кислоты и их применение |
USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
WO2010123369A1 (en) | 2009-04-24 | 2010-10-28 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
EP2251437B1 (en) | 2009-05-13 | 2013-12-04 | Hannelore Breitenbach-Koller | Method for identifying compounds that control the translational activity of ribosomal proteins in differential mRNA expression |
US8247436B2 (en) | 2010-03-19 | 2012-08-21 | Novartis Ag | Pyridine and pyrazine derivative for the treatment of CF |
JP2015509922A (ja) | 2012-01-27 | 2015-04-02 | プロセンサ テクノロジーズ ビー.ブイ.Prosensa Technologies B.V. | デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
EP2928471B1 (en) | 2012-12-06 | 2020-10-14 | Celgene Quanticel Research, Inc. | Histone demethylase inhibitors |
CN103074346A (zh) * | 2013-01-22 | 2013-05-01 | 山西大学 | 一种用于促通读药物筛选的有限细胞株的建立方法 |
BR112016007255A2 (pt) | 2013-10-03 | 2017-09-12 | Moderna Therapeutics Inc | polinucleotídeos que codificam receptor de lipoproteína de baixa densidade |
TWI636978B (zh) * | 2014-03-06 | 2018-10-01 | 美商Ptc治療公司 | 1,2,4-二唑苯甲酸之鹽及醫藥組合物 |
JP6543268B2 (ja) * | 2014-04-15 | 2019-07-10 | バーテックス ファーマシューティカルズ インコーポレイテッドVertex Pharmaceuticals Incorporated | 嚢胞性線維症膜コンダクタンス調節因子が媒介する疾患を処置するための医薬組成物 |
JP2016155900A (ja) * | 2015-02-23 | 2016-09-01 | 株式会社フジミインコーポレーテッド | 研磨用組成物、研磨方法及び硬脆材料基板の製造方法 |
TW201729807A (zh) | 2015-10-30 | 2017-09-01 | Ptc治療公司 | 用於治療癲癇的方法 |
US10898483B2 (en) | 2015-12-23 | 2021-01-26 | Moonshot Pharma Llc | Methods for inducing an immune response by promoting premature termination codon read-through |
EP3512516A1 (en) * | 2016-09-13 | 2019-07-24 | Marco Cipolli | Method of treatment of shwachman-diamond syndrome |
JP2020525434A (ja) | 2017-06-22 | 2020-08-27 | ムーンショット ファーマ エルエルシー | アンレキサノクス及び免疫調節剤を含む組成物で癌を治療する方法 |
CN107916252B (zh) * | 2017-11-17 | 2021-01-22 | 首都儿科研究所 | 一株用于验证脊肌萎缩症的永生淋巴细胞株 |
EA202192767A1 (ru) * | 2019-04-10 | 2021-12-29 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Способ лечения опосредуемой нонсенс-мутацией мышечной дистрофии дюшенна у педиатрических пациентов |
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Also Published As
Publication number | Publication date |
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RU2008142957A (ru) | 2010-05-10 |
IL222557A0 (en) | 2012-12-31 |
BRPI0710213A2 (pt) | 2011-04-12 |
US20160199357A1 (en) | 2016-07-14 |
CN101505739A (zh) | 2009-08-12 |
CA2647903C (en) | 2016-12-13 |
RU2462246C2 (ru) | 2012-09-27 |
IL222557A (en) | 2016-09-29 |
SG170819A1 (en) | 2011-05-30 |
NZ596965A (en) | 2013-06-28 |
JP2009532368A (ja) | 2009-09-10 |
MX2008012515A (es) | 2008-11-19 |
IL194454A0 (en) | 2009-08-03 |
IN2014CN04406A (no) | 2015-09-04 |
WO2007117438A2 (en) | 2007-10-18 |
US9289398B2 (en) | 2016-03-22 |
AU2007235524A1 (en) | 2007-10-18 |
NZ571899A (en) | 2012-07-27 |
EP2007365A2 (en) | 2008-12-31 |
CA2647903A1 (en) | 2007-10-18 |
IL194454A (en) | 2013-01-31 |
EP2387995A1 (en) | 2011-11-23 |
JP5367563B2 (ja) | 2013-12-11 |
JP5798154B2 (ja) | 2015-10-21 |
US20110046136A1 (en) | 2011-02-24 |
AU2007235524B2 (en) | 2013-09-12 |
JP2013234189A (ja) | 2013-11-21 |
WO2007117438A3 (en) | 2008-10-23 |
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