Journals Higher Education
Important OUP's Response to COVID-19 Learn more



Published: 19 September 2013

320 Pages


ISBN: 9780199915866

Also Available As:


Bookseller Code (AU)

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

Also of Interest