Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

Metachromatic leukodystrophy is a lysosomal storage disease, which is characterized by
damage of the myelin sheath that covers most of nerve fibers of the central and peripheral …

Abnormalities in neuronal cholesterol homeostasis have been suspected or observed in
several neurodegenerative disorders including Alzheimer's disease, Parkinson's disease …

Neurological disorders–disorders of the brain, spine and associated nerves–are a leading
contributor to global disease burden with a shockingly large associated economic cost …

Clinical gene therapy has made important advances over the last decade. Among
neurological diseases, severe genetic neurodegenerative conditions have been the focus of …

The development of Alzheimer's disease (AD) is closely connected with cholesterol
metabolism. Cholesterol increases the production and deposition of amyloid-β (Aβ) peptides …

The identification of the most efficient method for whole central nervous system targeting that
is translatable to humans and the safest route of adeno-associated virus (AAV) …

Friedreich ataxia (FA) is a rare mitochondrial disease characterized by sensory and
spinocerebellar ataxia, hypertrophic cardiomyopathy, and diabetes, for which there is no …

O-sulfogalactosylceramide or sulfatide is a major component of the myelin sheath in the
central and peripheral nervous system. The examination of mice deficient in the sulfatide …

Adeno-associated viral vectors (AAVs) have demonstrated potential in applications for
neurologic disorders, and the discovery that some AAVs can cross the blood-brain barrier …