Category: News

Gene therapy of hemoglobinopathies: progress and future challenges.

Abstract Recently, gene therapy clinical trials have been successfully applied to hemoglobinopathies, such as sickle cell disease (SCD) and β-thalassemia. Among the great discoveries that led to the design of genetic approaches to cure these disorders is the discovery of the β-globin locus control region (LCR) and several associated transcription factors, which determine hemoglobin switching …

Analgesic management of uncomplicated acute sickle-cell pain crisis in pediatrics: a systematic review and meta-analysis.

OBJECTIVES: To capture evidence of the efficacy and safety of pharmacological analgesia for uncomplicated acute sickle-cell pain in pediatric patients compared to placebo. SOURCES OF DATA: Searches for key evidence were performed from March 1 to 31, 2018, for randomized controlled trials of pharmacological analgesia compared to placebo for uncomplicated acute sickle-cell pain in a …