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Small Molecule Therapy for Genetic Disease PDF
Edited by Jess G. (University of Michigan, Ann Arbor) Thoene
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Description
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.
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- Format:PDF
- Pages:18 Tables, unspecified; 4 Plates, unspecified; 27 Line drawings, unspecified
- Publisher:Cambridge University Press
- Publication Date:30/09/2010
- Category:
- ISBN:9780511910890
Information
-
Download Now
- Format:PDF
- Pages:18 Tables, unspecified; 4 Plates, unspecified; 27 Line drawings, unspecified
- Publisher:Cambridge University Press
- Publication Date:30/09/2010
- Category:
- ISBN:9780511910890