Abstract Background Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)
is derived from the bacterial innate immune system and engineered as a robust gene-editing …

Introduction Friedreich ataxia (FRDA) is a rare autosomal recessive degenerative disorder
characterized by ataxia, dysarthria, diabetes, cardiomyopathy, scoliosis, and occasionally …

Alzheimer's disease (AD) is the most prevalent cause of dementia; microglia have been
implicated in AD pathogenesis, but their role is still matter of debate. Our study showed that …

Friedreich's ataxia (FRDA) is an inherited, multisystemic disorder predominantly caused by
GAA hyper expansion in intron 1 of frataxin (FXN) gene. This expansion mutation …

Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the
blood and immune systems has placed them at the forefront of cell and gene therapy …

Friedreich ataxia (FRDA) is a rare genetic multisystem disorder caused by a pathological
GAA trinucleotide repeat expansion in the FXN gene. The numerous drawbacks of historical …

This narrative review aims at providing an update on the management of inherited
cerebellar ataxias (ICAs), describing main clinical entities, genetic analysis strategies and …

The conditions supporting the generation of microglia-like cells in the central nervous
system (CNS) after transplantation of hematopoietic stem/progenitor cells (HSPC) have …

Metachromatic leukodystrophy (MLD) is a rare genetic disorder caused by mutations in the
Arylsulfatase-A (ARSA) gene. The enzyme plays a key role in sulfatide metabolism in brain …

Most Friedreich ataxia (FRDA) cases are caused by the elongation of the GAA repeat (GAAr)
sequence in the first intron of the FXN gene, leading to a decrease of the frataxin protein …