In the last few decades, many pathogenic or likely pathogenic genetic mutations in over
hundred different genes have been described for non-ischemic, genetic cardiomyopathies …

Fabry disease is a multi-organ disease, caused by mutations in the GLA gene and leading to
a progressive accumulation of glycosphingolipids due to enzymatic absence or malfunction …

The coronavirus disease 2019 (COVID-19) pandemic with high infectivity and mortality has
caused severe social and economic impacts worldwide. Growing reports of COVID-19 …

Rare cardiovascular diseases (RCDs) refer to those cardiovascular diseases that display a
low prevalence as well as morbidity. Due to the vast variety of underlying genetic mutations …

Fabry disease (FD) is a lysosomal storage disorder resulting from mutations in the alpha-
galactosidase A (GLA) gene, characterized by pain, skin lesions, renal failure, and cardiac …

The late-onset type of Fabry disease (FD) with GLA IVS4+ 919G> A mutation has been
shown to lead to cardiovascular dysfunctions. In order to eliminate variations in other …

The major function of the lysosome is to degrade unwanted materials such as lipids,
proteins, and nucleic acids; therefore, deficits of the lysosomal system can result in improper …

Inherited cardiomyopathies are among the major causes of heart failure and associated with
significant mortality and morbidity. Currently, over 70 genes have been linked to the etiology …

Non-viral gene delivery holds promises for treating inherited diseases. However, the limited
cloning capacity of plasmids may hinder the co-delivery of distinct genes to the transfected …

Introduction Fabry disease (FD) results from pathogenic GLA variants, leading to a
deficiency in lysosomal α-galactosidase A (α-Gal A) and accumulation of the sphingolipid …