For families of the pediatric rare disease community, headlines drive elevated hope and excitement. But they also lead to heightened expectations and emotional strain for those facing more complex rare diseases — like the 89.6% of them that affect the nervous system … which are unlikely to be in line for the first generation of gene therapies. The headlines may lead families to misunderstand the technical and biological barriers of delivering gene therapies to the human brain ….
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The new Center for Pediatric CRISPR Cures is directing its first efforts toward developing personalized CRISPR on-demand treatments for severe pediatric conditions such as rare metabolic disorders and inborn errors of immunity, where patients can’t break down basic nutrients ….
Early progress in CRISPR gene therapies will help to standardize the delivery process and suggest how to streamline it to become more affordable and accessible. The same technology could be used to treat diseases with more complex biology — particularly the brain. But while many researchers and clinicians are working to develop gene therapies for neurological disorders, good science takes time.
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