Achieving sufficient delivery across the blood–brain barrier is a key challenge in the
development of drugs to treat central nervous system (CNS) disorders. This is particularly …

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 …

Crossing the blood–brain barrier in primates is a major obstacle for gene delivery to the
brain. Adeno-associated viruses (AAVs) promise robust, non-invasive gene delivery from the …

Adeno-associated viruses (AAVs) are commonly used for in vivo gene transfer.
Nevertheless, AAVs that provide efficient transduction across specific organs or cell …

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 development of gene therapies for the treatment of diseases of the central nervous
system has been hindered by the limited availability of adeno-associated viruses (AAVs) that …

In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-
associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential …

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 …

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) …

Neurological disorders affecting the central nervous system (CNS) are still incompletely
understood. Many of these disorders lack a cure and are seeking more specific and effective …