Inherited retinal diseases (IRDs) are a clinically complex and heterogenous group of visual
impairment phenotypes caused by pathogenic variants in at least 277 nuclear and …

Alternative splicing of pre-mRNA increases genetic diversity, and recent studies estimate
that most human multiexon genes are alternatively spliced. If this process is not highly …

Brain postnatal development is characterized by critical periods of experience-dependent
remodeling of neuronal circuits. Failure to end these periods results in neurodevelopmental …

Alteration of brain aerobic glycolysis is often observed early in the course of Alzheimer's
disease (AD). Whether and how such metabolic dysregulation contributes to both synaptic …

Presynaptic glutamate replenishment is fundamental to brain function. In high activity
regimes, such as epileptic episodes, this process is thought to rely on the glutamate …

Alzheimer's disease (AD) is characterized by synaptic failure, dendritic and axonal atrophy,
neuronal death and progressive loss of cognitive functions. It is commonly assumed that …

Nanomaterials are currently being developed for the specific cell/tissue/organ delivery of
genetic material. Nanomaterials are considered as non-viral vectors for gene therapy use …

Neurodegenerative disorders are a major public health problem because of the high
frequency of these diseases. Genome editing with the CRISPR/Cas9 system is making it …

Removal of introns from pre‐mRNA precursors (pre‐mRNA splicing) is a necessary step for
the expression of most genes in multicellular organisms, and alternative patterns of intron …

Inherited retinal diseases (IRDs) are both genetically and clinically highly heterogeneous
and have long been considered incurable. Following the successful development of a gene …