Inherited retinal dystrophies (IRDs) are a group of rare eye diseases caused by gene
mutations that result in the degradation of cone and rod photoreceptors or the retinal …

Background Neurodevelopmental disorders, as a class of diseases, have been particularly
difficult to treat even when the underlying cause (s), such as genetic alterations, are …

Vectors derived from adeno-associated viruses (AAVs) have become important gene
delivery tools for the treatment of many inherited ocular diseases in well-characterized …

Background The ability to deliver a gene of interest into a specific cell type is an essential
aspect of biomedical research. Viruses can be a useful tool for this delivery, particularly in …

Adeno-associated viral vectors (AAVs) are increasingly useful preclinical tools in
neuroscience research studies for interrogating cellular and neurocircuit functions and …

Gene therapy is a promising modality for the treatment of inherited and acquired
cardiovascular diseases. The identification of the molecular pathways involved in the …

Gene therapy has strong potential for treating a variety of genetic disorders, as
demonstrated in recent clinical trials. There is unfortunately no scarcity of disease targets …

The cerebellar involvement in cognitive functions such as attention, language, working
memory, emotion, goal-directed behavior and spatial navigation is constantly growing …

Recombinant adeno-associated virus (rAAV) holds promise as a gene therapy vector for a
multitude of genetic disorders such as hemophilia, cystic fibrosis, and the muscular …

Adeno-associated virus (AAV) has a single-stranded DNA genome encapsidated in a small
icosahedrally symmetric protein shell with 60 subunits. AAV is the leading delivery vector in …