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 …

Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

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

Late-stage retinal degenerative disease involving photoreceptor loss can be treated by
optogenetic therapy, cell transplantation and retinal prostheses. These approaches aim to …

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 …

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

Background/aims Voretigene neparvovec (VN) is the first and only subretinal gene therapy
approved by the Food and Drug Administration and European Medicines Agency. Real …

Inherited retinal diseases (IRDs) are a genetically variable collection of devastating
disorders that lead to significant visual impairment. Advances in genetic characterization …

Achromatopsia (ACHM), also known as rod monochromatism or total color blindness, is an
autosomal recessively inherited retinal disorder that affects the cones of the retina, the type …

In 2001, the first large animal was successfully treated with a gene therapy that restored its
vision. Lancelot, the Briard dog that was treated, suffered from a human childhood blindness …