Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

Viral vectors have been used for a broad spectrum of gene therapy for both acute and
chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor …

Inherited retinal diseases (IRD) are a leading cause of blindness in the working age
population and in children. The scope of this review is to familiarise clinicians and scientists …

Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of
disorders characterised by photoreceptor degeneration or dysfunction. These disorders …

A resurgence of interest and investment in the field of gene therapy, driven in large part by
advances in viral vector technology, has recently culminated in United States Food and Drug …

Purpose.: An often overlooked prerequisite to cone photoreceptor gene therapy
development is residual photoreceptor structure that can be rescued. While advances in …

Due to improved phenoty** and genetic characterization, the field of 'incurable'and
'blinding'inherited retinal diseases (IRDs) has moved substantially forward. Decades of …

Leber congenital amaurosis (LCA) associated with retinal pigment epithelium-specific
protein 65 kDa (RPE65) mutations is a severe hereditary blindness resulting from both …

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 contributions of genetics research to the science of normal and defective color vision
over the previous few decades are reviewed emphasizing the developments in the 25years …