Article
Development of pathophysiologically relevant models of sickle cell disease and β-thalassemia for therapeutic studies.
Nature communications - 27 Feb 2024
Gupta Pragya, Goswami Sangam Giri, Kumari Geeta, Saravanakumar Vinodh, Bhargava Nupur, Rai Akhila Balakrishna, Singh Praveen, Bhoyar Rahul C, Arvinden V R, Gunda Padma, Jain Suman, Narayana Vanya Kadla, Deolankar Sayali C, Prasad T S Keshava, Natarajan Vivek T, Scaria Vinod, Singh Shailja, Ramalingam Sivaprakash
Abstract excerpt
Ex vivo cellular system that accurately replicates sickle cell disease and β-thalassemia characteristics is a highly sought-after goal in the field of erythroid biology. In this study, we present the generation of erythroid progenitor lines with sickle cell disease and β-thalassemia mutation using CRISPR/Cas9. The disease cellular models exhibit similar differentiation profiles, globin expression and proteome...
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