Inherited and age-related retinal degeneration is the hallmark of a large group of
heterogeneous diseases and is the main cause of untreatable blindness today. Genetic …

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

Purpose To characterize the genetic landscape of patients with suspected retinitis
pigmentosa (RP) in the Chinese population. Design Cohort study. Participants A total of …

Ciliopathies manifest from sensory abnormalities to syndromic disorders with multi-organ
pathologies, with retinal degeneration a highly penetrant phenotype. Photoreceptor cell …

Inherited retinal degeneration (RD) leads to the impairment or loss of vision in millions of
individuals worldwide, most frequently due to the loss of photoreceptor (PR) cells. Animal …

Major advances in the study of inherited retinal diseases (IRDs) have placed efforts to
develop treatments for these blinding conditions at the forefront of the emerging field of …

Gene augmentation and genome editing are promising strategies for the treatment of
monogenic inherited retinal diseases. Although gene augmentation treatments are …

Inherited retinal degenerations (IRDs) are a leading cause of blindness. Although gene-
supplementation therapies have been developed, they are only available for a small …

The use of RNA interference technology has proven to inhibit the expression of many target
genes involved in the underlying pathogenesis of several diseases affecting various …

Inherited retinal dystrophies (IRDs) are a clinically and genetically heterogeneous group of
disorders that often severely impair vision. Some patients manifest poor central vision as the …