Gene-Editing, Sickle Cell Treatment , Gets UK Approval .<br />Regulators in the United Kingdom have <br />authorized the world's first gene therapy <br />treatment for people with sickle cell disease.<br />Regulators in the United Kingdom have <br />authorized the world's first gene therapy <br />treatment for people with sickle cell disease.<br />'Time' reports that the decision could offer <br />relief for thousands of people in the U.K. <br />who suffer from the crippling disease.<br />On November 16, the Medicines and Healthcare <br />Regulatory Agency said it approved Casgevy, <br />which uses the gene-editing tool CRISPR.<br />The U.K. regulator approved Casgevy for the <br />treatment of patients over the age of 12 <br />with sickle cell disease and thalassemia. .<br />The future of life-changing <br />cures resides in CRISPR-based<br />(gene-editing) technology, Dr. Helen O'Neill, University College London, via 'Time'.<br />The use of the word ‘cure’ <br />in relation to sickle cell disease <br />or thalassemia has, up until <br />now, been incompatible. , Dr. Helen O'Neill, University College London, via 'Time'.<br />Dr. Helen O'Neill, of University College <br />London, called the MHRA's approval of Casgevy , “a positive moment in history.”.<br />'Time' reports that sickle cell disease can block blood <br />flow and result in excruciating pain, organ damage, <br />strokes and other serious health problems.<br />The new treatment is currently under review by <br />the United States Food and Drug Administration, <br />which is expected to deliver a decision next month.<br />High costs have caused experts to express <br />concerns that gene therapy treatments could be <br />financially out of reach for most people.
