With the improvement of gene vectors, the rise of chimeric antigen receptor T cell
immunotherapy and breakthroughs in the genome editing technology, gene therapy had …

Pompe disease is a rare and deadly muscle disorder. As a clinical entity, the disease has
been known for over 75 years. While an optimist might be excited about the advances made …

Dravet syndrome (DS) is an intractable developmental and epileptic encephalopathy
caused largely by de novo variants in the SCN1A gene, resulting in haploinsufficiency of the …

Since the discovery of the first microRNA (miRNA), the exploration of miRNA biology has
come to a new era in recent decades. Monumental studies have proven that miRNAs can be …

Nanoparticle drug delivery systems have proved anti-tumor effects; however, they are not
widely used in tumor therapy due to insufficient ability to target specific sites, multidrug …

Background: Gene therapy cannot be yet considered a far perspective, but a tangible
therapeutic option in the field of retinal diseases. Although still confined in experimental …

Gene therapies aim to deliver a therapeutic payload to specified tissues with underlying
protein deficiency. Since the 1990s, gene therapies have been explored as potential …

Alzheimer's disease (AD) is a progressive and irreversible neurodegenerative disorder
clinically characterized by cognitive impairment, abnormal behavior, and social deficits …

Pompe disease is an inherited neuromuscular disorder caused by deficiency of the
lysosomal enzyme acid alpha-glucosidase (GAA). The most severe form is infantile-onset …

Mycoplasma gallisepticum (MG) is an important avian pathogen that causes significant
economic losses in the poultry industry. Surprisingly, the limited protection and adverse …