WO2014067038A1 - Point repère, préparation et méthode de traitement de carence en adsl chez l'être humain - Google Patents
Point repère, préparation et méthode de traitement de carence en adsl chez l'être humain Download PDFInfo
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- WO2014067038A1 WO2014067038A1 PCT/CN2012/082887 CN2012082887W WO2014067038A1 WO 2014067038 A1 WO2014067038 A1 WO 2014067038A1 CN 2012082887 W CN2012082887 W CN 2012082887W WO 2014067038 A1 WO2014067038 A1 WO 2014067038A1
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- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
-
- 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
- C12Y—ENZYMES
- C12Y401/00—Carbon-carbon lyases (4.1)
- C12Y401/01—Carboxy-lyases (4.1.1)
- C12Y401/01021—Phosphoribosylaminoimidazole carboxylase (4.1.1.21)
Definitions
- ADSL enzyme adenyl succinate lyase
- PAICS phosphoribosylaminoimidazole succinocarboxamide synthetase /phosphoribosylaminoimidazole carboxylase
- SAICAR phosphoribosylaminoimidazole succinocarboxamide synthetase
- AIRc 5-aminoimidazole ribonucleotide carboxylase
- Another object of the present invention is to provide a preparation for treating human ADSL deficiency.
- Figure 4 shows the relative increase in length of nematode RNA interference.
- a method of treating human ADSL deficiency comprising administering to a patient a formulation comprising at least one of the following:
- nematodes In the nematode, many genes play an important role in embryonic development and growth of the worm. About 60% of the genes in the nematode The gene is homologous to human genes, and therefore, nematodes are also widely used in the study of human genetic metabolic diseases [Kuwabara, P.E., and O'Neil, N. (2001). The Use of functional genomics in C. elegans for studying human development and Journal of Inherited Metabolic Disease 24, 127-138.] . At present, people mainly use nematodes to study apoptosis, neurodevelopment, behavioral biology, etc., but the use of nematodes as model organisms to study human ADSL deficiency has not been reported.
- the nematode gene ADSL ( R06C7.5a ) is homologous to the human gene ADSL .
- nematodes can be used to study ADSL deficiency, and study the phenotype of nematode growth and development after genetic ADSL deficiency. influences.
- Transfer culture of nematodes from a piece of NGM-OP50 containing a large number of nematodes A small amount of agar medium was cut out from the plate with a sterile blade, transferred to a new NGM-OP50 plate, and cultured in a 16 °C incubator.
- HT115-L4440 from the LB-tetra+ plate.
- HT115-L4440-1100 HT115 monoclonal to 2ml LB-Cb+ liquid medium cultured overnight at 37 °C shaker.
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Pain & Pain Management (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Abstract
L'invention concerne des points repères qui permettent de traiter une carence en ADSL chez l'être humain, les points repères comportant un gène PAICS, un ARNm de gène PAICS ou de protéine PAICS. L'invention concerne également l'utilisation de gène PAICS, d'ARNm de gène PAICS ou de protéine PAICS dans le traitement de carence en ADSL chez l'homme.
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| PCT/CN2012/082887 WO2014067038A1 (fr) | 2012-10-29 | 2012-10-29 | Point repère, préparation et méthode de traitement de carence en adsl chez l'être humain |
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| PCT/CN2012/082887 WO2014067038A1 (fr) | 2012-10-29 | 2012-10-29 | Point repère, préparation et méthode de traitement de carence en adsl chez l'être humain |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| WO2014067038A1 true WO2014067038A1 (fr) | 2014-05-08 |
Family
ID=50626280
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PCT/CN2012/082887 WO2014067038A1 (fr) | 2012-10-29 | 2012-10-29 | Point repère, préparation et méthode de traitement de carence en adsl chez l'être humain |
Country Status (1)
| Country | Link |
|---|---|
| WO (1) | WO2014067038A1 (fr) |
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018029430A1 (fr) * | 2016-08-10 | 2018-02-15 | Assistance Publique-Hopitaux De Paris (Ap-Hp) | Utilisation d'un inhibiteur de la synthese de novo des purines, dans le traitement du deficit en adenylosuccinate lyase |
| WO2018059395A1 (fr) * | 2016-09-29 | 2018-04-05 | 广州君赫生物科技有限公司 | Composés affaiblissant l'activité de la saicar synthase, et applications |
| WO2018059213A1 (fr) * | 2016-09-29 | 2018-04-05 | 广州君赫生物科技有限公司 | Composés interférant avec la synthèse de saicar, et applications |
| US11517541B2 (en) | 2017-04-20 | 2022-12-06 | Geneheal Biotechnology Co., Ltd. | Applications of spermidine and its derivatives |
| US11684593B2 (en) | 2017-04-20 | 2023-06-27 | Geneheal Biotechnology Co., Ltd. | Applications of spermine and its derivative in preparation of antitumor drug |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011034421A1 (fr) * | 2009-09-16 | 2011-03-24 | Stichting Het Nederlands Kanker Instituut | Gènes cibles fra-1 utilisés comme cibles médicamenteuses pour le traitement du cancer |
| CN101999002A (zh) * | 2008-02-04 | 2011-03-30 | 彼帕科学公司 | 诊断和治疗parp-介导的疾病的方法 |
-
2012
- 2012-10-29 WO PCT/CN2012/082887 patent/WO2014067038A1/fr active Application Filing
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101999002A (zh) * | 2008-02-04 | 2011-03-30 | 彼帕科学公司 | 诊断和治疗parp-介导的疾病的方法 |
| WO2011034421A1 (fr) * | 2009-09-16 | 2011-03-24 | Stichting Het Nederlands Kanker Instituut | Gènes cibles fra-1 utilisés comme cibles médicamenteuses pour le traitement du cancer |
Cited By (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018029430A1 (fr) * | 2016-08-10 | 2018-02-15 | Assistance Publique-Hopitaux De Paris (Ap-Hp) | Utilisation d'un inhibiteur de la synthese de novo des purines, dans le traitement du deficit en adenylosuccinate lyase |
| WO2018059395A1 (fr) * | 2016-09-29 | 2018-04-05 | 广州君赫生物科技有限公司 | Composés affaiblissant l'activité de la saicar synthase, et applications |
| WO2018059213A1 (fr) * | 2016-09-29 | 2018-04-05 | 广州君赫生物科技有限公司 | Composés interférant avec la synthèse de saicar, et applications |
| WO2018059211A1 (fr) * | 2016-09-29 | 2018-04-05 | 广州君赫生物科技有限公司 | Nouvelles applications de spermine et de dérivés de celle-ci |
| WO2018059396A1 (fr) * | 2016-09-29 | 2018-04-05 | 广州君赫生物科技有限公司 | Composés affaiblissant l'activité de la saicar synthase, et applications |
| US20190209499A1 (en) * | 2016-09-29 | 2019-07-11 | Geneheal Biotechnology Co., Ltd. | Compounds weakening saicar synthetase activity and applications |
| US10709676B2 (en) | 2016-09-29 | 2020-07-14 | Geneheal Biotechnology Co., Ltd. | Applications of spermine and derivatives thereof |
| US11766412B2 (en) | 2016-09-29 | 2023-09-26 | Geneheal Biotechnology Co., Ltd. | Methods of treating or alleviating adenylosuccinatelyase (ADSL) deficiency using spermidine or a pharmaceutically acceptable salt of spermidine |
| US11517541B2 (en) | 2017-04-20 | 2022-12-06 | Geneheal Biotechnology Co., Ltd. | Applications of spermidine and its derivatives |
| US11684593B2 (en) | 2017-04-20 | 2023-06-27 | Geneheal Biotechnology Co., Ltd. | Applications of spermine and its derivative in preparation of antitumor drug |
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