Beta testing: preclinical genome editing in β-globin disorders
نویسندگان
چکیده
The β-globin disorders β-thalassemia and sickle cell disease have been at the forefront of gene therapy development from its very inception. Owing to their frequency, severity and exceptionally well characterized molecular pathology, and to the availability of hematopoietic stem and progenitor cells as substrate for therapies, these disorders promise both fast insights into new methodologies and eventual return on investment. Accordingly, β-globinopathies are also a favorite subject of the nascent field of genome editing and its most pioneering approaches to achieving therapeutic functional correction of gene expression. Be it by mutation-specific correction, modulation of disease modifiers or site-specific gene addition, genome editing of β-globinopathies has already delivered significant insights into the design of synthetic nucleases and the suitability of different correction strategies. This Expert Insight reviews recent progress in the application of gene-editing tools and different model systems towards the establishment of new therapies for β-globin disorders.
منابع مشابه
Nuclease-mediated gene editing by homologous recombination of the human globin locus
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