MicroRNA-regulated viral vectors for gene therapy

Barriers and recent advances in non-viral vectors targeting the lungs for cystic fibrosis gene therapy

Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in CFTR genes that affect chloride ion channel. The CF is a good nominee for gene therapy as the asymptomatic carriers are phenotypically normal, and the desired cells are accessible for vector delivery. Gene therapy shows promising effects involving the correction of gene or replacement of the mutant gene with the func...

Physiologically-Regulated Expression Vectors for Gene Therapy

Gene-replacement gene therapy has been under development for a number of years. In spite of the large amount of research invested into developing gene therapy for the treatment of recessive genetic disorders only a limited number of patients world-wide have received the benefits. In addition, several high profile adverse events in gene therapy trials have lead to an increasing awareness of the ...

18 Viral Vectors for Gene Therapy

Adeno-associated viral vectors for gene transfer and gene therapy.

Adeno-associated virus (AAV) is a defective, non-pathogenic human parvovirus that depends for growth on coinfection with a helper adenovirus or herpes virus. Recombinant adeno-associated viruses (rAAVs) have attracted considerable interest as vectors for gene therapy. In contrast to other gene delivery systems, rAAVs lack all viral genes and show long-term gene expression in vivo without immune...

Viral vectors in pancreatic cancer gene therapy

Min Li *, Joel A. Rodriguez, William E. Fisher, Xiaoliu Zhang, Changyi Chen and Qizhi Yao * Molecular Surgeon Research Center, Elkins Pancreas Center, Michael E. DeBakey Department of Surgery, Department of Molecular Virology and Microbiology, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, Texas 77030 ______________________________________________________________________...