Liver fibrosis is a wound-healing response that results from various chronic damages. If the
causes of damage are not removed or effective treatments are not given in a timely manner …

Lentiviral vectors (LVs) are important for cell therapy because of their capacity to stably
modify the genome after integration. This study describes a novel and relatively simple …

Recently, clinical trials of adeno-associated virus-mediated replacement therapy have
suggested long-term therapeutic effects for several genetic diseases of the liver, including …

Advances in cellular engineering, as well as gene, and cell therapy, may be used to produce
human tissues with programmable genetically enhanced functions designed to model …

The multi-administration of recombinant adeno-associated virus (rAAV) is limited largely by
immunological barriers. Herein, a novel strategy, named rAAV pseudo-lipid nanoparticle …

The adeno-associated virus (AAV) vector is one of the most advanced platforms for gene
therapy because of its low immunogenicity and non-pathogenicity. The concentrations of …

Alpha-1 antitrypsin deficiency (AATD) is characterized by both chronic lung disease due to
loss of wild-type AAT (M-AAT) antiprotease function and liver disease due to toxicity from …

Sexual dimorphism creates different biological and cellular activities and selective
regulation mechanisms in males and females, thus generating differential responses in …

Mucopolysaccharidosis type I-Hurler (MPS IH) is a neurodegenerative lysosomal storage
disorder (LSD) caused by inherited defects of the α-L-iduronidase (IDUA) gene. Current …

Advances in cellular engineering, as well as gene, and cell therapy, may be used to produce
human tissues with programmable genetically enhanced functions designed to model …