Optogenetic approaches promise to revolutionize neuroscience by using light to manipulate
neural activity in genetically or functionally defined neurons with millisecond precision …

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

Recessive Stargardt disease (STGD1) is an inherited blinding disorder caused by mutations
in the Abca4 gene. ABCA4 is a flippase in photoreceptor outer segments (OS) that …

Over the last decade, huge progress has been made in the understanding of the molecular
mechanisms underlying inherited retinal dystrophy (IRD), as well as in the development and …

From Bench to Bedside''was held June 15 and 16, 2012, at the University of Colorado
Anschutz Medical Campus in Aurora, Colorado. The SERC provided a diverse group of …

This review offers the basics of lentiviral vector technologies, their advantages and pitfalls,
and an overview of their use in the field of ophthalmology. First, the description of the global …

Photoreceptors are specialized neurons of the retina that receive nursing from the adjacent
retinal pigment epithelium (RPE). Frequent in the elderly, photoreceptor loss can originate …

Introduction: Cystic fibrosis (CF) remains a life-threatening genetic disease, with few
clinically effective treatment options. Gene therapy and gene editing strategies offer the …

Gene therapy strategies for the treatment of inherited retinal diseases have made major
advances in recent years. This review focuses on adeno-associated viral (AAV) vector …

Gliosis is a complex process comprising upregulation of intermediate filament (IF) proteins,
particularly glial fibrillary acidic protein (GFAP) and vimentin, changes in glial cell …