Loss-of-function heterozygous mutations in GRN, the gene encoding progranulin (PGRN),
were identified in patients with frontotemporal lobar degeneration (FTLD) almost two …

Recombinant adeno-associated viruses (AAVs) are commonly used gene delivery vehicles
for neuroscience research. They have two engineerable features: the capsid (outer protein …

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 virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Adeno-associated viral (AAV) vectors are attractive tools for central nervous system (CNS)
gene therapy because some vectors can cross the blood-brain barrier (BBB), allowing them …

Therapeutic payload delivery to the central nervous system (CNS) remains a major
challenge in gene therapy. Recent studies using function-driven evolution of adeno …

Intracerebral vector delivery in nonhuman primates has been a major challenge. We report
successful blood-brain barrier opening and focal delivery of adeno-associated virus …

Different glial cell types are found throughout the central (CNS) and peripheral nervous
system (PNS), where they have important functions. These cell types are also involved in …

Stroke may cause severe death and disability but many clinical trials have failed in the past,
partially because the lack of an effective method to regenerate new neurons after stroke. In …

The blood-brain barrier (BBB) presents a major challenge for delivering large molecules to
study and treat the central nervous system. This is due in part to the scarcity of targets known …