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 …

Over the past 20 years, there has been tremendous progress in retinal gene therapy. The
safety and efficacy results in one early-onset severe blinding disease may lead to the first …

Amyotrophic lateral sclerosis (ALS) is a fatal and incurable neurodegenerative disease
characterized by the progressive loss of motor neurons in the spinal cord and brain. In …

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 …

Dual leucine zipper kinase (DLK) has been implicated in cell death signaling secondary to
axonal damage in retinal ganglion cells (RGCs) and other neurons. To better understand the …

Usher syndrome type III (USH3) characterized by progressive loss of vision and hearing is
caused by mutations in the clarin-1 gene (CLRN1). Clrn1 knockout (KO) mice develop hair …

There is much debate on the adeno-associated virus (AAV) serotype that best targets
specific retinal cell types and the route of surgical delivery—intravitreal or subretinal. This …

Recombinant adeno-associated virus (rAAV) vectors are proving to be a reliable gene
transfer system for several clinical applications, with an increasing body of evidence …

Adeno-associated virus (AAV) is the most common vector for clinical gene therapy of the
CNS. This popularity originates from a high safety record and the longevity of transgene …

One of the great advantages of the retina as a target tissue for gene delivery is the wide
array of genetic tools that have been developed in the past decade. This includes a variety …