Genome Editing

for Sickle Cell Disease

Client: NHLBI


Sickle cell disease is a genetic disorder caused by mutations in the beta globin gene, leading to faulty hemoglobin protein, called hemoglobin S. Genetic therapies are approaches to treat genetic disorders by adding new DNA or changing existing DNA. Nucleus highlights how researchers are testing ways to use genome editing technologies to treat sickle cell disease.

See also



Decoding Cancer Immunology


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