Genome engineering through CRISPR-Cas9 is a revolutionary approach with diverse applications ranging from gene discovery to crop improvement, exploration of disease pathophysiology, development of disease models, identification of drug targets, etc. It has also been applied as a therapeutic intervention for treating various genetic diseases. Correction of a faulty gene or introduction of a desirable mutation has been shown to reverse genetic disease-associated symptoms for currently incurable and complicated pathophysiologies.

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Sickle Cell Disease is monogenic in nature with a well-characterized point mutation in the HBB gene. It has one of the highest incidence rates in the Indian population. especially in low socio-economic status communities. The current treatment approaches are restricted to the use of Hydroxyurea, blood transfusions, and eventually, stem cell transplants. A crucial way to tackle this growing number of patients is the identification of carriers and counseling of patients besides finding an effective, robust, and cheap gene editing method. In this direction, our lab is working on developing base editors and CRISPR-Cas-based treatment options for SCD and other hemoglobinopathies like thalassemia. Our lab focuses on engineering highly specific and effective CRISPR-Cas9-based genome editing tools, mainly with the use of enhanced FnCas9 and base editing for developing curative therapies for Sickle Cell Disease, Duchenne Muscular Dystrophy, FENIB, etc. The lab is also exploring if the enhanced FnCas9 versions exhibit higher HDR efficiency for the same system