Recent advancements in genome editing, particularly CRISPR-Cas9 technology, are paving the way for potential treatments for severe brain disorders. Although gene therapies for other organs have shown success, the brain presents unique challenges due to its protective barriers. There is a growing interest in identifying genetic causes of brain disorders, leading to increased support from families seeking answers and treatments. Researchers are optimistic about the future of brain-targeted gene editing, with hopes that trials will begin soon.
The data have never looked so good. This is less and less science fiction, and closer to reality.
The question that I hear from our families is, 'It was done so quickly for him. What's taking us so long?'
People are starting to now find out that their child's seizures, for example, are related to particular genetic mutations.
Researchers are searching for similar particles that can selectively target the brain, which is surrounded by a defensive barrier.
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