A YOUNG AMERICAN BECOMES THE FIRST PERSON IN THE WORLD TO RECOVER FROM SICKLE DISEASE! UN JEUNE AMÉRICAIN DEVIENT LA PREMIÈRE PERSONNE AU MONDE À GUÉRIR DE LA DREPANOCYTOSE!
English version A young American by the name of Daniel Cressy, has become the first person in the world to be cured from sickle cell disease, thanks to a new therapy called ex vivo gene-editing. This therapy pharmacological mechanism doesn't involve cutting out the sickle mutation itself and Instead, utilizes CRISPR-Cas9 to induce a strategic genetic workaround. The therapy relies on reactivating the production of fetal hemoglobin (HbF), which has a high oxygen affinity and does not polymerize like mutated adult sickle hemoglobin (HbS). The Target: The CRISPR-Cas9 machinery is directed to the erythroid-specific enhancer region of the BCL11A gene. The Edit: It introduces double-strand breaks that are repaired via non-homologous end joining (NHEJ), resulting in indels that disrupt the enhancer. The Result: BCL11A is a transcription factor that normally represses gamma-globin (HbF) expression after birth. By knocking down BCL11A expression specific...