Fast Five Quiz: Genomic Medicine — CRISPR Gene Editing

Kiran Musunuru, MD, PhD


December 23, 2021

Gene-editing technology is potentially useful for treating genetically inherited conditions because all of these diseases at their core have a fundamental aberration in the DNA.

Blood disorders in general, both malignant and nonmalignant, represent "model diseases" because the fundamental alterations in their DNA underlie the conditions and symptoms. Sickle cell anemia and alpha and beta thalassemia are examples. Recognizing the fundamental elements in those conditions has enabled the ability to track specific regions in the DNA and to guide the deletion or the replacement of certain nucleotide bases, correcting the mutations or altering other DNA regions in ways that can compensate for the mutations.

Learn more about clinical applications for CRISPR.


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