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

Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno …

Prime editing enables a wide variety of precise genome edits in living cells. Here we use
protein evolution and engineering to generate prime editors with reduced size and improved …

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 …

Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery
vehicles resulting in three US Food and Drug Administration (FDA) and one European …

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 …

Recombinant adeno-associated virus (AAV) is a promising delivery vehicle for in vivo gene
therapy and has been widely used in> 200 clinical trials globally. There are already several …

Abstract Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic
diseases caused by functional defects in specific lysosomal proteins. The lysosome is a …

Lysosomal storage diseases (LSDs) are a group of metabolic inborn errors caused by
defective enzymes in the lysosome, resulting in the accumulation of undegraded substrates …

Recombinant adeno-associated virus (rAAV) vectors could be manufactured by plasmid
transfection into human embryonic kidney 293 (HEK293) cells or baculovirus infection of …