Gene Therapy for Rare Neurological Disorders

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 range of other gene therapies for rare neurologic diseases are at various stages of development. Future development of gene editing technologies promises further to broaden the potential for more patients with these disorders to benefit from innovative therapies.

Automated summary

There are over 7,000 individually rare diseases that collectively affect millions of people globally. Neurologic single-gene disorders are particularly common among them. Recent advancements in recombinant adeno-associated virus vectors have paved the way for gene therapies for rare neurologic diseases, including FDA-approved treatments for inherited retinal dystrophy and spinal muscular atrophy. Further development of gene editing technologies holds the promise of expanding innovative therapies to benefit more patients with these disorders.