Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

Alzheimer's disease (AD) is a neurodegenerative disorder with devastating symptoms,
including memory impairments and cognitive deficits. Hallmarks of AD pathology are …

Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

Deep brain stimulation (DBS) has evolved considerably over the past 4 decades. Although it
has primarily been used to treat movement disorders such as Parkinson's disease, essential …

Abstract Alzheimer's Disease (AD), the most prevalent neurodegenerative disease of aging,
affects one in eight older Americans. Nearly all drug treatments tested for AD today have …

This review provides an overview of recent developments in “chemical virology.” Viruses, as
materials, provide unique nanoscale scaffolds that have relevance in chemical biology and …

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 …

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 …

It has been well established that neuronal loss within the cholinergic nucleus basalis of
Meynert (nbM) correlates with cognitive decline in dementing disorders such as Alzheimer's …

Alzheimer disease (AD) represents an oncoming epidemic that without an effective
treatment promises to exact extraordinary financial and emotional burdens (Apostolova …