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酶替代疗法:问题与前景

Enzyme-replacement therapy: problems and prospects.

作者信息

Rademaker B, Raber J

机构信息

Department of Pharmacology, Duphar BV, Weesp, The Netherlands.

出版信息

Pharm Weekbl Sci. 1989 Oct 20;11(5):137-45. doi: 10.1007/BF01959460.

DOI:10.1007/BF01959460
PMID:2687794
Abstract

Several diseases can, at least in theory, be treated by the administration of an enzyme, the deficiency of which is the cause of the disease. Various attempts have been made to correct enzymatic deficiencies responsible for the clinical manifestation of diseases for which prevention cannot be achieved by modification of the diet or by supportive therapy with drugs. Except for treating certain digestive disorders, enzyme-replacement therapy has not yet found a broad application. In this review article a compilation is given of the problems and prospects of enzyme-replacement therapy in diseases caused by the deficiency of an enzyme.

摘要

至少在理论上,有几种疾病可以通过给予一种酶来治疗,而该酶的缺乏正是导致这些疾病的原因。人们已经进行了各种尝试,以纠正那些导致疾病临床表现的酶缺乏症,而这些疾病无法通过调整饮食或药物支持疗法来预防。除了治疗某些消化系统疾病外,酶替代疗法尚未得到广泛应用。在这篇综述文章中,我们总结了酶替代疗法在由酶缺乏引起的疾病中的问题和前景。

相似文献

1
Enzyme-replacement therapy: problems and prospects.酶替代疗法:问题与前景
Pharm Weekbl Sci. 1989 Oct 20;11(5):137-45. doi: 10.1007/BF01959460.
2
Molecular pathologies of and enzyme replacement therapies for lysosomal diseases.溶酶体疾病的分子病理学及酶替代疗法
CNS Neurol Disord Drug Targets. 2006 Aug;5(4):401-13. doi: 10.2174/187152706777950738.
3
Enzyme replacement and enhancement therapies for lysosomal diseases.溶酶体疾病的酶替代和增强疗法。
J Inherit Metab Dis. 2004;27(3):385-410. doi: 10.1023/B:BOLI.0000031101.12838.c6.
4
Role of Nanotechnology for Enzyme Replacement Therapy in Lysosomal Diseases. A Focus on Gaucher's Disease.纳米技术在溶酶体疾病酶替代疗法中的作用。聚焦戈谢病。
Curr Med Chem. 2016;23(9):929-52. doi: 10.2174/0929867323666160210130608.
5
Enzymatic therapy of peptic ulcer and digestive disorders.消化性溃疡和消化系统疾病的酶疗法。
Am J Gastroenterol. 1960 Jan;33:80-9.
6
Red cell enzyme deficiencies: from genetic basis to gene transfer.红细胞酶缺乏症:从遗传基础到基因转移
Semin Hematol. 1998 Apr;35(2):126-35.
7
[Enzyme therapy of digestive disorders].[消化系统疾病的酶疗法]
Medizinische. 1956 May 26(21):795-8.
8
Enzyme-nanoparticle conjugates for biomedical applications.用于生物医学应用的酶-纳米颗粒缀合物
Methods Mol Biol. 2011;679:165-82. doi: 10.1007/978-1-60761-895-9_14.
9
The enzyme as drug: application of enzymes as pharmaceuticals.酶作为药物:酶在制药领域的应用。
Curr Opin Biotechnol. 2003 Aug;14(4):444-50. doi: 10.1016/s0958-1669(03)00092-2.
10
[Enzyme therapy in digestive disorders].[消化系统疾病的酶疗法]
Med Klin. 1959 Jan 23;54(4):140-3.

本文引用的文献

1
Orthotopic Liver Transplantation in Children With Hepatic-Based Metabolic Disease.患有肝脏基础代谢疾病儿童的原位肝移植
Transplant Proc. 1983 Mar;15(1):1284-1287.
2
[Treatment of hemophilia B with a plasma fraction rich in antihemophilic B factor (P.P.B.)].
Pathol Biol. 1959 Dec;7:2507-13.
3
Immunological studies on modified enzymes. I. soluble L-asparaginase/mouse albumin copolymer with enzyme activity and substantial loss of immunogenicity.修饰酶的免疫学研究。I. 具有酶活性且免疫原性大幅降低的可溶性L-天冬酰胺酶/小鼠白蛋白共聚物
Int Arch Allergy Appl Immunol. 1981;64(1):11-8.
4
Effect of cimetidine and sodium bicarbonate on pancreatic replacement therapy in cystic fibrosis.西咪替丁和碳酸氢钠对囊性纤维化患者胰腺替代治疗的影响。
Gut. 1980 Sep;21(9):778-86. doi: 10.1136/gut.21.9.778.
5
Lectin-induced accumulation of large lysosomes in cultured fibroblasts.凝集素诱导培养的成纤维细胞中大型溶酶体的积累。
Exp Cell Res. 1980 Jul;128(1):133-42. doi: 10.1016/0014-4827(80)90396-1.
6
Maturation of alpha-L-iduronidase in cultured human fibroblasts.α-L-艾杜糖醛酸酶在培养的人成纤维细胞中的成熟过程。
J Biol Chem. 1981 Mar 25;256(6):3044-8.
7
Properties of two urate oxidases modified by the covalent attachment of poly(ethylene glycol).通过聚乙二醇共价连接修饰的两种尿酸氧化酶的性质
Biochim Biophys Acta. 1981 Aug 13;660(2):293-8. doi: 10.1016/0005-2744(81)90173-x.
8
Clinical evaluation of the hepatitis safety of a beta-propiolactone/ultraviolet treated factor IX concentrate (PPSB).β-丙内酯/紫外线处理的凝血因子IX浓缩剂(PPSB)肝炎安全性的临床评估
Thromb Res. 1982 Oct 1;28(1):75-83. doi: 10.1016/0049-3848(82)90035-4.
9
Hollow-fiber reactors containing mammalian arginase: an approach to enzyme replacement therapy.含有哺乳动物精氨酸酶的中空纤维反应器:一种酶替代疗法的方法。
Biochem Med. 1982 Feb;27(1):46-55. doi: 10.1016/0006-2944(82)90007-2.
10
Infantile hypophosphatasia diagnosed at 4 months and surviving at 2 years.婴儿期低磷酸酯酶症于4个月时确诊,患儿2岁时存活。
Helv Paediatr Acta. 1982;37(1):49-58.