Amyotrophic lateral sclerosis (ALS) has historically posed unique challenges for gene-
therapy-based approaches, due to a paucity of therapeutic targets as well as the difficulty of …

Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) primarily affect the
motor and frontotemporal areas of the brain, respectively. These disorders share clinical …

A GGGGCC24+ hexanucleotide repeat expansion (HRE) in the C9ORF72 gene is the most
common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia …

Frontotemporal dementia (FTD) encompasses a spectrum of clinical syndromes
characterized by progressive executive, behavioural and language dysfunction. The various …

In recent years, gene therapy has been raising hopes toward viable treatment strategies for
rare genetic diseases for which there has been almost exclusively supportive treatment. We …

Since the discovery of Cu/Zn superoxide dismutase (SOD1) gene mutation, in 1993, as the
first genetic abnormality in amyotrophic lateral sclerosis (ALS), over 50 genes have been …

Amyotrophic lateral sclerosis (ALS) is a fatal progressive neurodegenerative disease
characterized by a permanent degeneration of both upper and lower motor neurons. Many …

RNA therapy is a treatment that regulates cell proteins and cures diseases by affecting the
metabolism of mRNAs in cells, which has cut a figure in the studies on various incurable …

CRISPR-Cas9-mediated homology-directed repair (HDR) is a versatile platform for creating
precise site-specific DNA insertions, deletions, and substitutions. These precise edits are …

RNA interference (RNAi) technology has been used for almost two decades to study gene
functions and in therapeutic approaches. It uses cellular machinery and small, designed …