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 …

The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-
associated (Cas) system provides a groundbreaking genetic technology that allows …

Gene correction is a valuable strategy for treating inherited retinal degenerative diseases, a
major cause of irreversible blindness worldwide. Single gene defects cause the majority of …

Motivation CRISPR/Cas9 is driving a broad range of innovative applications from basic
biology to biotechnology and medicine. One of its current issues is the effect of off-target …

The potential of personalised genome editing reaching the clinic has come to light due to
advancements in the field of gene editing, namely the development of CRISPR/Cas9. The …

The fruit fly Drosophila melanogaster has served as an excellent model to study and
understand the genetics of many human diseases from cancer to neurodegeneration …

Degenerative retinal diseases such as retinitis pigmentosa (RP) and Leber's congenital
amaurosis (LCA) may lead to blindness without effective treatment. With the rapid …

Tanycytes are highly specialized bipolar ependymal cells that line the ventrolateral wall and
the floor of the third ventricle in the brain and form a blood-cerebrospinal fluid barrier at the …

Retinitis pigmentosa (RP) is a heterogeneous group of hereditary retinal degenerations for
which there is currently no cure. Studies investigating the use of gene therapy, gene editing …

Curing a genetic disease by repairing the underlying genetic defect is a fascinating concept
that has been addressed so far by gene compensation therapy. For this, a functional copy of …