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Chapter 49 - Stem Cell Transplantation: Clinical Potential in Treating Fetal Genetic Disease

from Fetal Stem Cell Transplantation

Published online by Cambridge University Press:  21 October 2019

By
Åsa Ekblad  and
Cecilia Götherström
Edited by
Mark D. Kilby ,
Anthony Johnson  and
Dick Oepkes
Mark D. Kilby
Affiliation:
University of Birmingham
Anthony Johnson
Affiliation:
University of Texas Medical School at Houston
Dick Oepkes
Affiliation:
Leids Universitair Medisch Centrum
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Summary

Inherited severe genetic diseases are rare but devastating disorders associated with vast morbidity and mortality. Currently there is no cure for many of these diseases, but merely pharmacological therapies replacing the missing gene product or inhibiting the progressing pathology of the disease. Stem cell transplantation has the possibility to restore the defect permanently and hematopoietic stem cell transplantation (HSCT) is today used postnatally to cure sickle-cell disease, severe combined immunodeficiency (SCID), and other immune disorders [1, 2]. However, the pathology is progressing with sustained morbidity during fetal life and the disease may already be manifested at birth.

Type
Chapter
Information
Fetal Therapy
Scientific Basis and Critical Appraisal of Clinical Benefits
 , pp. 512 - 519
Publisher: Cambridge University Press
Print publication year: 2020

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