Gene Therapy for Rare Neurological Disorders.

Abstract excerpt

There are over 7,000 diseases that are individually rare, but collectively affect millions of people worldwide. They are very commonly neurologic single-gene disorders. Recent advances in recombinant adeno-associated virus vectors have enabled breakthroughs, including US Food and Drug Administration (FDA)-approved gene therapies for inherited retinal dystrophy due to RPE65 mutation and spinal muscular atrophy. A...

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