Journals Higher Education



Published: 04 September 2013

320 Pages

6-1/8 x 9-1/4 inches

ISBN: 9780199915866

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Bookseller Code (05)

Seeking Cures

Design of Therapies for Genetically Determined Diseases

Moyra Smith, MD, PhD

  • Outline of the processes typical for identifying disease-modifying therapies
  • Examples of newer therapeutic approaches in use or under investigation to treat lysosomal storage diseases, inborn errors of metabolism, mitochondrial functional defects, and specific monogenic diseases
  • Considers the aspects of treatment at the cellular level
  • Provides a glimpse at the pursuit-and future-of personalized medicine

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