Seeking cures : design of therapies for genetically determined diseases /

Seeking cures : design of therapies for genetically determined diseases /

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Bibliographic Details
Author / Creator:	Smith, Moyra, author.
Imprint:	Oxford : Oxford University Press, 2014.
Description:	1 online resource
Language:	English
Subject:	Genetic disorders -- Diagnosis. Genetic disorders -- Treatment. Genetic Diseases, Inborn -- therapy. Genetic Therapy. Genetic disorders -- Diagnosis. Genetic disorders -- Treatment.
Format:	E-Resource Book
URL for this record:	http://pi.lib.uchicago.edu/1001/cat/bib/11754159

Hidden Bibliographic Details
ISBN:	9780199915873 0199915873 9780199326051 0199326053 9780199915866 0199915865
Notes:	Includes bibliographical references and index. Print version record.
Summary:	"Progress in molecular and cellular biology has greatly enhanced our ability to accurately diagnose diseases that are caused by gene mutations, changes in genome structures, and altered gene expression; increased emphasis is now placed on translational research the clinical treatment of these genetically determined diseases. Seeking Cures outlines the progress and implications of science's quest to identify therapeutic targets and initiate novel treatments at the gene, RNA, protein, and physiological levels. Also considered are aspects of treatment at the cellular level (e.g., those with hematopoietic stem cells or induced pluripotent stem cells)"--Publisher's description.
Other form:	Print version: Smith, Moyra. Seeking cures 9780199915866

Table of Contents:

Introduction and history
Therapy : design and general approaches
Inborn errors of metabolism : progress in diagnosis and treatment
Lysosomal storage diseases and therapies
Mitochondrial function, defects and approaches to treatment
Protein misfolding, endoplasmic reticulum stress and pathogenesis of disease
Transporters and solute carriers : passage of molecules across membranes
Advances in therapy for monogenic diseases
Identifying therapeutic targets in complex, multifactorial diseases
Approaches to cancer treatment
Gene based molecular therapies
Stem cells and pluripotent stem cells.