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Casgevy CRISP-R Sickle Cell Outcomes by iapodaca is a document available to read on EtoBox.

What is Casgevy CRISP-R Sickle Cell Outcomes about?

Sickle cell disease (SCD) is a prevalent monogenic blood disorder primarily caused by a single mutation, with CRISPR technology emerging as a promising treatment option. Recent trials of Casgevy, the first approved CRISPR therapy, show over 90% success in alleviating symptoms and increasing fetal hemoglobin in patients. However, challenges such as potential off-target effects and high treatment costs remain significant barriers to accessibility.

Author

iapodaca

Language

EN