Small Molecule Therapy for Genetic Disease
Thoene J.
ISBN: 9780521517812
Vydavatelství: Cambridge University Press
Rok vydání: 2010
Vazba: Hardback
Počet stran: 238
Původní cena: 3 783 Kč
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3 216 Kč(t.j. po slevě 15%)
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Katalogová cena: 89.99 GBP
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Thoene summarises the substantial work that has been accomplished in the treatment of inborn errors of metabolism with simple molecules. This handbook will enable interested clinician scientists to rapidly survey the field, thus ascertaining what has been done as well as future directions for therapeutic research. Its important introductory chapters discuss the infrastructure of the field. The book closely analyses the cofactors used to augment the function of defective enzymes and the compounds that are able to utilise an alternative pathway in order to avoid the consequences of the metabolic block present in the patient. Among other therapies, the authors discuss the use of zinc and tetrathiomolybdate to treat Wilson's disease and the use of cysteamine to treat nephropathic cystinosis.