In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

The neurodegenerative disease spinal muscular atrophy (SMA) is caused by deficiency in
the survival motor neuron (SMN) protein. Currently approved SMA treatments aim to restore …

Twenty-five years ago, the underlying genetic cause for one of the most common and
devastating inherited diseases in humans, spinal muscular atrophy (SMA), was identified …

Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …

Treatment of certain central nervous system disorders, including different types of cerebral
malignancies, is limited by traditional oral or systemic administrations of therapeutic drugs …

Gene therapy is rapidly emerging as a powerful therapeutic strategy for a wide range of
neurodegenerative disorders, including Alzheimer's disease (AD), Parkinson's disease (PD) …

Spinal muscular atrophy (SMA) is the most frequent lethal genetic neurodegenerative
disorder in infants. The disease is caused by low abundance of the survival of motor neuron …

Several attempts have been made to discover the ideal vector for gene therapy in central
nervous system (CNS). Adeno-associated viruses (AAVs) are currently the preferred vehicle …

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 …