Opinion: Celebrating a new, faster path to gene-editing medicines on demand
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In May, news broke<\/a> of a biomedical first: the on-demand design and clinical use of a personalized gene editor for a baby boy born with a rare, severe genetic condition. At the STAT Summit in October, the child\u2019s treating physician from the Children\u2019s Hospital of Philadelphia (CHOP) and the lead on developing the gene editor for him shared the stage with him and his parents. The video<\/a> of a happily squirming 1-year-old brought tears of joy to the eyes of many.<\/p>\n A sobering reminder of the long road ahead toward more such videos was shared<\/a> in these pages by Celena Lozano, whose son lives with a no less rare and severe genetic condition called PURA syndrome. Concerned over false hope inadvertently created by exuberant media coverage of the success story, she wrote, \u201cfamilies of the rare disease community deserve to have a better understanding of when their child might be next.\u201d<\/p>\n