Gene therapies are currently one of the most investigated therapeutic modalities in both the
preclinical and clinical settings and have shown promise in treating a diverse spectrum of …

Recombinant adeno-associated viruses (AAVs) are commonly used gene delivery vehicles
for neuroscience research. They have two engineerable features: the capsid (outer protein …

The GBA gene encodes for the lysosomal enzyme glucocerebrosidase (GCase), which
maintains glycosphingolipid homeostasis. Approximately 5–15% of PD patients have …

Mitochondrial diseases are clinically heterogeneous disorders caused by a wide spectrum
of mutations in genes encoded by either the nuclear or the mitochondrial genome …

In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

INTRODUCTION The capacity of a tissue to regenerate itself rests on the potential of its
resident cells to replace cells lost to injury. Some tissues, such as skin or intestine, do this …

The development of gene therapies for the treatment of diseases of the central nervous
system has been hindered by the limited availability of adeno-associated viruses (AAVs) that …

Reactive oxygen species (ROS) derived from oxygen-dependent mitochondrial metabolism
are the essential drivers of cardiomyocyte (CM) cell-cycle arrest in adulthood. Mitochondria …

Spinal muscular atrophy type 1 (SMA1) is a debilitating neurodegenerative disease resulting
from survival motor neuron 1 gene (SMN1) deletion/mutation. Onasemnogene abeparvovec …

The recent years saw the advent of promising preclinical strategies that combat the
devastating effects of a spinal cord injury (SCI) that are progressing towards clinical trials …