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加罗德的先见之明;我们的后见之明。

Garrod's foresight; our hindsight.

作者信息

Scriver C R

机构信息

Department of Human Genetics, McGill University and McGill University Health Centre, Montreal, Quebec, Canada.

出版信息

J Inherit Metab Dis. 2001 Apr;24(2):93-116. doi: 10.1023/a:1010351630856.

DOI:10.1023/a:1010351630856
PMID:11405353
Abstract

Archibald Edward Garrod introduced a paradigm, new for its day, in medicine: Biochemistry is dynamic and different from the static nature of organic chemistry. It led him to think about metabolic pathways and to recognize that variation in Mendelian heredity could explain an 'inborn error of metabolism'. At the time, Garrod had no idea about the nature of a gene. Genes are now well understood, genomes are being described for one organism after another (including H. sapiens) and it is understood that genomes 'speak biochemistry (not phenotype)'. Accordingly, in the era of genomics, biochemistry and physiology become the bases of functional genomics and it is possible to appreciate why 'nothing in biology makes sense without evolution' (and nothing in medicine will make sense without biology). Mendelian, biochemical and molecular genetics together have revealed what lies behind the four canonical inborn errors described by Garrod (albinism, alkaptonuria, cystinuria and pentosuria). Both older and newer ideas in genetics, new tools for applying them, and renewed respect for the clinician-scientist will enhance our understanding of the human biological variation that accounts for variant states of health and overt disease; an 'unsimple' phenotype (phenylketonuria) is used to illustrate in some detail. What can be known and what ought to be done with knowledge about human genetics to benefit individuals, families and communities (society) is both opportunity and challenge.

摘要

阿奇博尔德·爱德华·加罗德在医学领域引入了一种在当时全新的范式:生物化学是动态的,与有机化学的静态性质不同。这使他开始思考代谢途径,并认识到孟德尔遗传中的变异可以解释“先天性代谢缺陷”。当时,加罗德对基因的本质一无所知。如今,基因已被充分了解,一个又一个生物体(包括智人)的基因组正在被描绘出来,并且人们明白基因组“讲述的是生物化学(而非表型)”。因此,在基因组学时代,生物化学和生理学成为了功能基因组学的基础,并且我们能够理解为什么“生物学中没有任何东西在没有进化的情况下是有意义的”(而医学中没有任何东西在没有生物学的情况下是有意义的)。孟德尔遗传学、生物化学遗传学和分子遗传学共同揭示了加罗德所描述的四种典型先天性代谢缺陷(白化病、尿黑酸尿症、胱氨酸尿症和戊糖尿症)背后的原因。遗传学领域的新旧思想、应用这些思想的新工具以及对临床科学家的重新尊重,将增进我们对导致健康变异状态和显性疾病的人类生物变异的理解;文中详细阐述了一种“不简单”的表型(苯丙酮尿症)。关于人类遗传学的知识,我们能够知晓什么以及应该如何利用这些知识来造福个人、家庭和社区(社会),这既是机遇也是挑战。

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Garrod's Croonian Lectures (1908) and the charter 'Inborn Errors of Metabolism': albinism, alkaptonuria, cystinuria, and pentosuria at age 100 in 2008.加罗德的克罗尼安讲座(1908年)与“先天性代谢缺陷”宪章:2008年白化病、尿黑酸尿症、胱氨酸尿症和戊糖尿症诞生100周年
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本文引用的文献

1
Detection of amino-acids in urine and other fluids.尿液及其他体液中氨基酸的检测。
Lancet. 1946 Nov 2;2(6427):637-9. doi: 10.1016/s0140-6736(46)90088-8.
2
Aminoacid metabolism in cystinuria.胱氨酸尿症中的氨基酸代谢
Q J Med. 1951 Jul;20(79):205-19.
3
The nature of the defect in tyrosine metabolism in alcaptonuria.黑尿症中酪氨酸代谢缺陷的本质。
Chemical individuality: concept and outlook.
化学个性:概念与展望。
J Inherit Metab Dis. 2008 Oct;31(5):630-40. doi: 10.1007/s10545-008-0995-6. Epub 2008 Oct 7.
4
Legacies of Garrod's brilliance. One hundred years--and counting.加罗德卓越成就的遗产。一百年——且仍在延续。
J Inherit Metab Dis. 2008 Oct;31(5):574-9. doi: 10.1007/s10545-008-0985-8. Epub 2008 Oct 5.
5
After the genome--the phenome?基因组之后——表型组?
J Inherit Metab Dis. 2004;27(3):305-17. doi: 10.1023/B:BOLI.0000031100.26546.6e.
6
Does hereditary metabolic disease modulate senescence and ageing?遗传性代谢疾病是否会调节衰老过程?
J Inherit Metab Dis. 2002 May;25(3):235-51. doi: 10.1023/a:1015650517020.
J Biol Chem. 1958 Jan;230(1):251-60.
4
Phenotypes and genotypes in cystinuria.胱氨酸尿症的表型与基因型
Ann Hum Genet. 1955 Aug;20(1):57-91. doi: 10.1111/j.1469-1809.1955.tb01278.x.
5
Molecular structure of nucleic acids; a structure for deoxyribose nucleic acid.核酸的分子结构;脱氧核糖核酸的一种结构。
Nature. 1953 Apr 25;171(4356):737-8. doi: 10.1038/171737a0.
6
A structural hypothesis for BH4 responsiveness in patients with mild forms of hyperphenylalaninaemia and phenylketonuria.关于轻度高苯丙氨酸血症和苯丙酮尿症患者对四氢生物蝶呤反应性的结构假说。
J Inherit Metab Dis. 2001 Apr;24(2):213-30. doi: 10.1023/a:1010371002631.
7
The role of chaperone-assisted folding and quality control in inborn errors of metabolism: protein folding disorders.伴侣蛋白辅助折叠和质量控制在先天性代谢缺陷:蛋白质折叠障碍中的作用。
J Inherit Metab Dis. 2001 Apr;24(2):189-212. doi: 10.1023/a:1010319001722.
8
Structural and functional analysis of mutations in alkaptonuria.黑尿症中突变的结构与功能分析
Hum Mol Genet. 2000 Sep 22;9(15):2341-50. doi: 10.1093/oxfordjournals.hmg.a018927.
9
Defective folding and rapid degradation of mutant proteins is a common disease mechanism in genetic disorders.突变蛋白的折叠缺陷和快速降解是遗传疾病中常见的疾病机制。
J Inherit Metab Dis. 2000 Jul;23(5):441-7. doi: 10.1023/a:1005663728291.
10
Mouse as the measure of man?以鼠量人?
Trends Genet. 2000 Jul;16(7):283-7. doi: 10.1016/s0168-9525(00)02039-4.