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 …

Cancers are defined by genetic defects, which underlines the prospect of using gene
therapy in patient care. During the past decade, CRISPR technology has rapidly evolved …

Designing artificial viral vectors (AVVs) programmed with biomolecules that can enter
human cells and carry out molecular repairs will have broad applications. Here, we describe …

Recombinant adeno-associated viruses (rAAVs) are a preferred vector system in clinical
gene transfer. A fundamental challenge to formulate and deliver rAAVs as stable and …

rAAV vectors vary in their effectiveness between species, making it difficult to predict clinical
outcomes. Here authors show that AAV capsid proteins influence the vector epigenomic …

Although more adeno-associated virus AAV-based drugs enter the clinic, vector tissue
tropism remains an unresolved challenge that limits its full potential despite that the tissue …

Accelerating the scale up of adeno-associated virus (AAV) manufacture is highly desirable
to meet the increased demand for gene therapies. However, the development of …

Gene therapies offer promising therapeutic alternatives for many disorders that currently lack
efficient treatment options. Due to their chemical nature and physico-chemical properties …

The advent of optogenetic tools has had a profound impact on modern neuroscience
research, revolutionizing our understanding of the brain. These tools offer a remarkable …

Recombinant AAV vectors have been, or are currently in use, in 331 Phase I/II/III clinical
trials in a number of human diseases, and in some cases, remarkable clinical efficacy has …