Gene therapy

Abstract

The integrity of the human genome is essential for maintaining the well being of an individual. Mutations, either caused by extrinsic or intrinsic means, that alters the genetic code can lead to genetic diseases, including, but not limited to, immature aging, developmental disorder, neurological disorder, cancer, etc. Traditional therapeutics may be able to treat or attenuate the condition of the genetic disease, but the mutated gene is still maintained in the patient's genome and the root of the problem is not addressed. Gene therapy, or genetic replacement therapy, aims to treats the disease at the genetic level, either by introducing a wild-type sequence gene over the defective one or a gene that encodes a therapeutic protein. There have been great challenges in developing gene therapy treatment in a safe, effective and specialized way for treating different genetic diseases. We will discuss (1) what diseases are considered to be suitable for gene therapy, (2) currently available methods used in gene delivery, (3) the choice of different viral and non-viral gene delivery tools and how they can be introduced into the target tissues or organs, (4) the safety aspects of gene therapy and (5) challenges yet to be solved in increasing the success rate of gene therapy. We hope to introduce to the readers the latest advancement of the gene therapy field and further discuss challenges and difficulties in having a successful outcome of gene therapy.