CRISPR systems mediate adaptive immunity in bacteria and archaea through diverse
effector mechanisms and have been repurposed for versatile applications in therapeutics …

The presence of the blood-brain barrier (BBB) remains a challenge in the treatment of
central nervous system (CNS) diseases, as it hinders the infiltration of many therapeutic …

Delivering genes to and across the brain vasculature efficiently and specifically across
species remains a critical challenge for addressing neurological diseases. We have evolved …

Despite major advancements in gene therapy technologies, there are no approved gene
therapies for diseases which predominantly effect the brain. Adeno-associated virus (AAV) …

Display of short peptides on the surface of adeno-associated viruses (AAVs) is a powerful
technology for the generation of gene therapy vectors with altered cell specificities and/or …

Gene therapy is making a comeback. With its twin promise of targeting disease etiology and
'long-term correction', gene-based therapies (defined here as all forms of genome …

Adeno-associated virus (AAV) vector, an excellent gene therapy vector, has been widely
used in the treatment of various central nervous system (CNS) diseases. Due to the …

Gene therapy may constitute a promising alternative to conventional pharmacological tools
and surgeries for epilepsy. For primary epilepsy, a single variant leading to a significant …

The corneal endothelium is critical for maintaining corneal clarity by mediating hydration
through barrier and pump functions. Progressive loss of corneal endothelial cells during …

Neuropsychiatric phenotypes have long been known to be influenced by heritable risk
factors, directly confirmed by the past decade of genetic studies that have revealed specific …