Inherited retinal diseases (IRDs) comprise a heterogeneous group of genetic disorders
affecting the retina. Caused by mutations in over 300 genes, IRDs result in visual impairment …

The emergence of genetic engineering at the beginning of the 1970′ s opened the era of
biomedical technologies, which aims to improve human health using genetic manipulation …

Gene therapy using recombinant adeno‐associated virus (rAAV) vectors to treat blinding
retinal dystrophies has become clinical reality. Therapeutically impactful targeting of …

There has been great progress in ocular gene therapy, but delivery of viral vectors to the
retinal pigmented epithelium (RPE) and retina can be challenging. Subretinal injection, the …

Understanding how the brain captures transient experience and converts it into long lasting
changes in neural circuits requires the identification and investigation of the specific …

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 …

Inherited retinopathies (IR) are common untreatable blinding conditions. Most of them are
inherited as monogenic disorders, due to mutations in genes expressed in retinal …

Widespread gene transfer to the retina is challenging as it requires vector systems to
overcome physical and biochemical barriers to enter and diffuse throughout retinal tissue …

The retina has been at the forefront of translational gene therapy. Proof-of-concept that gene
therapy could restore vision in a large animal led to the initiation of the first successful …

Within the next decade, we will see many gene therapy clinical trials for eye diseases, which
may lead to treatments for thousands of visually impaired people around the world. To target …