BRPI0411291A - esfingomielinase acida humana modificada tendo atividade aumentada e métodos para a fabricação da mesma - Google Patents
esfingomielinase acida humana modificada tendo atividade aumentada e métodos para a fabricação da mesmaInfo
- Publication number
- BRPI0411291A BRPI0411291A BRPI0411291-1A BRPI0411291A BRPI0411291A BR PI0411291 A BRPI0411291 A BR PI0411291A BR PI0411291 A BRPI0411291 A BR PI0411291A BR PI0411291 A BRPI0411291 A BR PI0411291A
- Authority
- BR
- Brazil
- Prior art keywords
- residue
- activity
- methods
- acid sphingomyelinase
- manufacture
- Prior art date
Links
- 230000000694 effects Effects 0.000 title abstract 5
- 238000000034 method Methods 0.000 title abstract 3
- 101000785978 Homo sapiens Sphingomyelin phosphodiesterase Proteins 0.000 title abstract 2
- 102000048866 human SMPD1 Human genes 0.000 title abstract 2
- 238000004519 manufacturing process Methods 0.000 title abstract 2
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 abstract 4
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 abstract 4
- 125000003396 thiol group Chemical group [H]S* 0.000 abstract 2
- 108090000790 Enzymes Proteins 0.000 abstract 1
- 102000004190 Enzymes Human genes 0.000 abstract 1
- 208000014060 Niemann-Pick disease Diseases 0.000 abstract 1
- 210000004899 c-terminal region Anatomy 0.000 abstract 1
- 238000007385 chemical modification Methods 0.000 abstract 1
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 abstract 1
- 235000018417 cysteine Nutrition 0.000 abstract 1
- 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 abstract 1
- 230000007812 deficiency Effects 0.000 abstract 1
- 238000006471 dimerization reaction Methods 0.000 abstract 1
- 230000007515 enzymatic degradation Effects 0.000 abstract 1
- 230000002255 enzymatic effect Effects 0.000 abstract 1
- 230000004048 modification Effects 0.000 abstract 1
- 238000012986 modification Methods 0.000 abstract 1
- 238000002741 site-directed mutagenesis Methods 0.000 abstract 1
Classifications
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Psychology (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
"ESFINGOMIELINASE ACIDA HUMANA MODIFICADA TENDO ATIVIDADE AUMENTADA E MéTODOS PARA A FABRICAçãO DA MESMA". Deficiências na atividade enzimática da esfingomielinase ácida (ASM) resultam na doença de Niemann-Pick. Uma variedade de modificações que eliminam a atividade do grupo de tiol livre do resíduo de cisteína do terminal C, resultam todas em um aumento substancial da atividade específica da enzima. Os métodos usados para a alteração da atividade desse resíduo incluem a mutagenese direcionada ao sítio para cancelar ou alterar o resíduo, degradação enzimática da ASM para a remoção do resíduo, dimerização da ASM promovida com cobre (através dos resíduos terminais de cisteína) e modificação química do grupo de tiol livre nesse resíduo.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US47812403P | 2003-06-12 | 2003-06-12 | |
| PCT/US2004/018515 WO2004111198A2 (en) | 2003-06-12 | 2004-06-10 | Modified human acid sphingomyelinase having increased activity, and methods for making the same |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BRPI0411291A true BRPI0411291A (pt) | 2006-08-01 |
Family
ID=33551809
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BRPI0411291-1A BRPI0411291A (pt) | 2003-06-12 | 2004-06-10 | esfingomielinase acida humana modificada tendo atividade aumentada e métodos para a fabricação da mesma |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US7527956B2 (pt) |
| EP (1) | EP1639085A2 (pt) |
| JP (1) | JP2007518396A (pt) |
| CN (1) | CN101027388A (pt) |
| BR (1) | BRPI0411291A (pt) |
| CA (1) | CA2528427A1 (pt) |
| IL (1) | IL172416A0 (pt) |
| WO (1) | WO2004111198A2 (pt) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2158322T3 (pl) | 2007-06-06 | 2017-10-31 | Genzyme Corp | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| US8742079B2 (en) | 2007-08-20 | 2014-06-03 | Protalix Ltd. | Saccharide-containing protein conjugates and uses thereof |
| CA2773133A1 (en) | 2009-08-28 | 2011-03-03 | Edward H. Schuchman | Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency |
| US9194011B2 (en) | 2009-11-17 | 2015-11-24 | Protalix Ltd. | Stabilized alpha-galactosidase and uses thereof |
| EP2665814B1 (en) | 2011-01-20 | 2017-05-17 | Protalix Ltd. | Nucleic acid construct for expression of alpha-galactosidase in plants and plant cells |
| CN107058531A (zh) * | 2017-04-01 | 2017-08-18 | 杭州艾迪康医学检验中心有限公司 | 一种检测尼曼‑匹克病smpd1基因突变的试剂盒和方法 |
| AU2022214529A1 (en) * | 2021-02-01 | 2023-08-03 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of niemann pick type a disease |
| TW202403043A (zh) * | 2022-03-18 | 2024-01-16 | 美商健臻公司 | 重組人類酸性鞘磷脂酶醫藥組合物及方法 |
-
2004
- 2004-06-10 BR BRPI0411291-1A patent/BRPI0411291A/pt not_active Application Discontinuation
- 2004-06-10 EP EP04754947A patent/EP1639085A2/en not_active Ceased
- 2004-06-10 US US10/864,981 patent/US7527956B2/en not_active Expired - Lifetime
- 2004-06-10 CN CNA2004800162354A patent/CN101027388A/zh active Pending
- 2004-06-10 WO PCT/US2004/018515 patent/WO2004111198A2/en not_active Ceased
- 2004-06-10 CA CA002528427A patent/CA2528427A1/en not_active Abandoned
- 2004-06-10 JP JP2006533700A patent/JP2007518396A/ja not_active Withdrawn
-
2005
- 2005-12-06 IL IL172416A patent/IL172416A0/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CA2528427A1 (en) | 2004-12-23 |
| US7527956B2 (en) | 2009-05-05 |
| WO2004111198A2 (en) | 2004-12-23 |
| WO2004111198A3 (en) | 2007-05-03 |
| IL172416A0 (en) | 2006-04-10 |
| CN101027388A (zh) | 2007-08-29 |
| US20050169906A1 (en) | 2005-08-04 |
| JP2007518396A (ja) | 2007-07-12 |
| EP1639085A2 (en) | 2006-03-29 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| PT93082A (pt) | Processo de producao de um derivado de activador de plaminogenio tissular, de plasmideo de expressao e de medicamento contendo o referido derivado | |
| BR9914746A (pt) | Polinucleotìdeos otimizados em vegetaiscodificando proteìnas pesticidas deaproximadamente 15 kda e de aproximadamente 45kda | |
| Tan et al. | Unveiling the elusive and exotic: Venomics of the Malayan blue coral snake (Calliophis bivirgata flaviceps) | |
| BR0213540A (pt) | Compostos, método para o tratamento de enfermidades ou distúrbios em um paciente e composição farmacêutica que compreende os mesmos | |
| BRPI9912227A (pt) | método para diagnóstico precoce in vitro de carcinomas e de seus estágios iniciais e kit para realização do método. | |
| BR0116370A (pt) | Composto, composição farmacêutica, uso de um composto, e, método de tratamento de uma doença ou condição mediada por hppar em um paciente. | |
| WO2007087367A3 (en) | Methods and compositions for modulating the mobilization of stem cells | |
| BR0201823A (pt) | Método para fornecimento de serviços diagnósticos clìnicos | |
| BRPI0413574A (pt) | aplicação de polipeptìdeos da famìlia cupredoxina na terapia do cáncer | |
| BR9816013A (pt) | Proteìna de ligação de interleucina-18 | |
| DE502005008015D1 (de) | Künstliches gelenkelement sowie ein damit ausgestattetes greifwerkzeug | |
| Vanhoof et al. | The forearm and hand musculature of semi‐terrestrial rhesus macaques (Macaca mulatta) and arboreal gibbons (Fam. Hylobatidae). Part I. Description and comparison of the muscle configuration | |
| JP7271180B2 (ja) | 神経損傷のための組み合わせ治療 | |
| BR9916323A (pt) | Inibidores da integrina alfavbeta6 | |
| BRPI0211635B8 (pt) | composto ciclo-hexil-sulfona, uso do mesmo, e, composição farmacêutica | |
| NO922573D0 (no) | Fremgangsmaate for aa paavise ben- og andre bindevevsforstyrrelser hos mennesker og dyr | |
| BR0001794A (pt) | Aplicação de fitosteróides (e seus isÈmeros), ácidofólico, cianocobalamina e piridoxina em fibrasdietéticas (alimentares) | |
| Alfaro-Chinchilla et al. | Venom composition, toxicity and cross-neutralization by PoliVal-ICP antivenom, of Mesoamerican jumping pitvipers genus Metlapilcoatlus (Viperidae: Crotalinae) | |
| WO2004111198A3 (en) | Modified human acid sphingomyelinase having increased activity, and methods for making the same | |
| Kearney | The phylogenetic position of Sineoamphisbaena hexatabularis reexamined | |
| El din Awad et al. | Comparative evaluation of d-glucosyl thiouronium, glucosylthio heterocycles, Daonil, and insulin as inhibitors for hepatic glycosidases | |
| PT1331940E (pt) | Utilizacao da pentraxina longa ptx3 para o tratamento de doencas provocadas por uma activacao alterada do factor de crescimento fgf-2 | |
| Hamrick | Functional osteology of the primate carpus with special reference to Strepsirhini | |
| Ogihara et al. | Carpal bones of N acholapithecus kerioi, a Middle Miocene Hominoid From N orthern K enya | |
| WO2003057719A3 (en) | Methods of modulating localization and physiological function of ip3 receptors |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B11A | Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing | ||
| B11Y | Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette] |