Non-viral Gene Therapy for Osteoarthritis

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...

Targeted, non-viral gene delivery for cancer gene therapy.

The ability to mediate targeted and specific delivery of therapeutics to cancer cells remains one of the most important hurdles in effectively treating cancer. This aspect also remains as one of the greatest limitations of gene therapy as well. Targeted vectors based on the use of DNA-binding agents attached to cell specific ligands or "molecular conjugates" were created with the goal of over-c...

Non-Viral Delivery Systems in Gene Therapy

Recent advances in molecular biology combined with the culmination of the Human Ge‐ nome Project [1] have provided a genetic understanding of cellular processes and disease pathogenesis; numerous genes involved in disease and cellular processes have been identi‐ fied as targets for therapeutic approaches. In addition, the development of high-throughput screening techniques (e.g., cDNA microarra...

Non-viral approaches to gene therapy.

There have been rapid advances in the development of non-viral techniques for gene transfer. Although viruses are highly evolved to infect mammalian cells, they have several limitations. The general theme is to mimic the advantageous components of viral systems whilst separating them from their limiting functions. The systems described here are broadly divided into true non-viral techniques and...

Gene Therapy for Human Osteoarthritis