Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

The human genetic code encrypted in thousands of genes holds the secret for synthesis of
proteins that drive all biological processes necessary for normal life and death. Though the …

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

Until recently, there was no approved treatment for a retinal degenerative disease.
Subretinal injection of a recombinant adeno-associated virus (AAV) delivering the normal …

Inherited retinal degenerations (IRDs) represent a diverse group of progressive, visually
debilitating diseases that can lead to blindness in which mutations in genes that are critical …

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

Retinal gene therapy is increasingly recognized as a novel molecular intervention that has
huge potential in treating common causes of blindness, the majority of which have a genetic …

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 …

Purpose To report the initial efficacy results of the Retina Implant Alpha AMS (Retina Implant
AG, Reutlingen, Germany) for partial restoration of vision in end-stage retinitis pigmentosa …

Leber congenital amaurosis type 10 (LCA10) is a severe inherited retinal dystrophy
associated with mutations in CEP290. The deep intronic c. 2991+ 1655A> G mutation in …