Current Advances and Challenges in Gene Therapies for Neurologic Disorders

Abstract excerpt

Over 300 million people globally are affected by rare diseases, many of which present predominantly with neurologic symptoms. Rare neurologic disorders pose significant diagnostic and therapeutic challenges including delayed diagnoses, limited treatment options, and a shortage of specialists. However, advancements in diagnostics, particularly next-generation sequencing and expansion of newborn screening, have...

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• Genetics and Neurodevelopmental Disorders
• Neurogenetic and Muscular Disorders Research
• Virus-based gene therapy research