Gene Therapy for Neurological Disorders
NEURODEGENERATIVE DISEASES Neurodegenerative diseases are a broad group of progressive disorders of the central nervous system (CNS) characterized by gradual loss of neuron structure and function . These disorders are typically chronic, incurable, and worsen with time. Common Examples of Neurodegenerative Diseases Disease Major Site Affected Key Clinical Feature ALS (Amyotrophic Lateral Sclerosis) Motor neurons Paralysis (Loss of voluntary motor function), muscle wasting (progressive weakness, thinning of muscle mass, and impaired mobility) FTD (Frontotemporal Dementia) Frontal (decision-making, planning, personality, voluntary motor control, and social behavior) & temporal lobes (Brain regions responsible for memory, language comprehension, and auditory processing) Personality & behavioral changes SCAs (Spinocerebellar Ataxias) Cerebellum Gait and coordination defects Huntington’s Disease (HD) Basal ganglia (A group of subcortical nuclei that regu...