Neurodegenerative diseases are prominent causes of pain, suffering, and death worldwide.
Traditional approaches modelling neurodegenerative diseases are deficient, and therefore …

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 …

The challenges in making animal models of complex human epilepsy phenotypes with
varied aetiology highlights the need to develop alternative disease models that can address …

Enhanced S-cone syndrome (ESCS) is caused by recessive mutations in the photoreceptor
cell transcription factor NR2E3. Loss of NR2E3 is characterized by repression of rod …

The CLN3 gene was identified over two decades ago, but the primary function of the CLN3
protein remains unknown. Recessive inheritance of loss of function mutations in CLN3 are …

Epilepsy is among the most common neurological disorders, affecting approximately 50
million people worldwide. Importantly, epilepsy is genetically and etiologically …

The neuronal ceroid lipofuscinoses (NCLs) are mostly seen as diseases affecting the central
nervous system, but there is accumulating evidence that they have co-morbidities outside …

CRISPR/Cas-mediated genome editing in human pluripotent stem cells (hPSCs) offers
unprecedented opportunities for develo** in vitro disease modeling, drug screening and …

Abstract National Eye Institute recently issued a new Strategic Plan outlining priority
research areas for the next 5 years. Starting cell source for deriving stem cell lines is as an …

Batten disease or neuronal ceroid lipofuscinosis (NCL) is a group of rare, fatal, inherited
neurodegenerative lysosomal storage disorders. Numerous genes (CLN1–CLN8, CLN10 …