Adeno-associated viruses (AAVs) are the safest and most effective gene delivery vehicles to
drive long-term transgene expression in gene therapy. While animal studies have shown …

Gene therapy has the potential to provide therapeutic benefit to millions of people with
neurodegenerative diseases through several means, including direct correction of …

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder
characterized by deficient synthesis of dopamine and serotonin. It presents in early infancy …

Parkinson's disease (PD) is the second-leading cause of dementia and is characterized by a
progressive loss of dopaminergic neurons in the substantia nigra alongside the presence of …

Alcohol use disorder (AUD) exacts enormous personal, social and economic costs globally.
Return to alcohol use in treatment-seeking patients with AUD is common, engendered by a …

Focused ultrasound (FUS)-induced with microbubbles (MBs) is a promising technique for
noninvasive opening of the blood-brain barrier (BBB) to allow targeted delivery of …

Nerve growth factor (NGF) gene therapy rescues and stimulates cholinergic neurons, which
degenerate in Alzheimer's disease (AD). In a recent clinical trial for AD, intraparenchymal …

The etiology of many neurological diseases affecting the central nervous system (CNS) is
unknown and still needs more effective and specific therapeutic approaches. Gene therapy …

We have previously shown that adeno-associated virus type 2 (AAV2) undergoes
anterograde axonal transport in rat and non-human primate brain. We screened other AAV …

Ischemic neuronal death causes serious lifelong neurological deficits; however, there is no
proven effective treatment that can prevent neuronal death after the ischemia. We …