CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease
Selami Demirci, Alexis Leonard, Khaled Essawi, John F. Tisdale
Abstract
enhancer editing in patients with β-hemoglobinopathies have demonstrated promising results, although follow-up is short and the number of patients treated to date is low. While practical, economic, and clinical challenges of genome editing are well recognized by the scientific community, potential solutions to overcome these hurdles are in development. Here, we review the recent progress and obstacles yet to be overcome for the most effective and feasible HbF reactivation practice using CRISPR-Cas9 genome editing as a curative strategy for patients with SCD.
Topics & Concepts
Genome editingFetal hemoglobinCRISPRCas9DiseaseEpigeneticsBiologyHemoglobinopathyEnhancerGenomeComputational biologyMedicineBioinformaticsGeneGeneticsFetusPregnancyGene expressionInternal medicineHemoglobinopathies and Related DisordersIron Metabolism and DisordersPrenatal Screening and Diagnostics