AbstractGene therapy is a therapeutic approach to deliver genetic material into cells to alter their function in entire organism. One promising form of gene delivery system (DDS) is liposomes. The success of liposome-mediated gene delivery is a multifactorial issue and well-designed liposomal systems might lead to optimized gene transfection particularly in vivo. Liposomal gene delivery systems...

AbstractGene therapy is a therapeutic approach to deliver genetic material into cells to alter their function in entire organism. One promising form of gene delivery system (DDS) is liposomes. The success of liposome-mediated gene delivery is a multifactorial issue and well-designed liposomal systems might lead to optimized gene transfection particularly in vivo. Liposomal gene delivery systems...

congenital myopathies are a clinically and genetically heterogeneous group of inherited muscle disorders characterized clinically by reduced fetal movements, hypotonia, weakness and developmental delay beginning at birth or in the first year of life. however, there can be a wide variation in clinical findings including mild and asymptomatic presentation to a severe form within each subtype with...

down syndrome (ds) is a birth defect with huge medical and social costs, caused by trisomy of whole or part of chromosome 21. it is the most prevalent genetic disease worldwide and the common genetic cause of intellectual disabilities appearing in about 1 in 400-1500 newborns. although the syndrome had been described thousands of years before, it was named after john langdon down who described ...

Gene therapy is a promising approach to treat intractable and refractory diseases at the genetic level. Basically, in gene therapy, target gene expression is induced by delivering foreign genes. Downregulation of target gene expression or gene silencing can also be performed using miRNA, siRNA or shRNA expression vectors [1]. Gene therapy is useful for both genetic and acquired diseases. For ge...

Currently, 33 gene-therapy drugs/products have been approved in the clinic. Over 3000 completed and ongoing clinical gene therapy trials reported worldwide. The development/maturation of tools for manipulation delivery, as well molecular advances diagnosis genetic diseases, played a central role therapy, which greatly revolutionized field. Versatile diverse manipulations deliveries, with possib...