Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Gene therapy reverses sickle cell phenotype in small study

Esrick EB et al. ASH 2018, Abstract 1023

Key clinical point: Gene therapy to induce the fetal form of hemoglobin reversed the symptoms of sickle cell disease in an adult patient.

Major finding: During 6 months of follow-up the patient did not experience sickle cell disease–related pain, respiratory events, or neurologic events, and did not have anemia.

Study details: A first-in-human study in seven adults with sickle cell disease.

Disclosures: Dr. Esrick reported receiving honoraria from Bluebird Bio, maker of the short hairpin RNA construct used in the trial.

Read the article here.

Citation:

Esrick EB et al. ASH 2018, Abstract 1023.