The impact of genetics on physiology and sports performance is one of the most debated
research aspects in sports sciences. Nearly 200 genetic polymorphisms have been found to …

The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

There are over 70 known lysosomal storage disorders (LSDs), most caused by mutations in
genes encoding lysosomal hydrolases. Central nervous system involvement is a hallmark of …

Hematopoietic stem and progenitor cell (HSPC) gene therapies have recently moved
beyond gene-addition approaches to encompass targeted genome modification or …

Metachromatic leukodystrophy, a fatal pediatric neurodegenerative lysosomal storage
disease caused by mutations in the arylsulfatase A (ARSA) gene, is characterized by …

The misuse of sport-related gene transfer methods in elite athletes is a real and growing
concern. The success of gene therapy in the treatment of hereditary diseases has been most …

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 …

Abstract N6‐methyladenosine (m6A) is the most abundant internal modification on
eukaryotic mRNA and has been implicated in a wide range of fundamental cellular …

Lysosomal storage disorders (LSD) are multisystemic progressive disorders caused by
genetic mutations involving lysosomal function. While LSDs are individually considered rare …

Background Viral vectors are attractive gene delivery vehicles because of their broad
tropism, high transduction efficiency, and durable expression. With no risk of integration into …