New Tech Research / Margot Harknett

Gene Therapy 2.0

Scientists have figured out a way to take an engineered virus and have it deliver healthy copies of a gene inside someone who has defective genes. They had complications at first, which halted the process when some patients were dying from the gene therapy. One problem they were having were that the viruses they developed to carry the cells took the genetic information to the wrong part of the gnome, leading to cancer causing genes to be activated. Another problem was the viruses would mess with the patient’s immune system, which would lead to organ failure. The emerging technology is called CRISPR-Cas9 gene editing and it has been used by researches at the University of Massachusetts (MIT) and Oregon Health Sciences University (OHSU) in successfully correcting hereditary tyrosinemia, a rare metabolic disease. CRISPR can be packaged into an AVV or adeno-associated virus, which is the most versatile gene delivery vehicle, and therefor scientists don’t need to send an entire gene because CRISPER is an editing system. This is a big step for gene therapy but it is just the beginning, researchers are looking into more complex common diseases such as Alzheimer’s, heart failure, and cancer that would require multiple genes that aren’t always involved in every case. A Harvard geneticist named George Church says that someday everyone may be able to use gene therapy to combat the effects of aging.

Gene therapy 2.0: Will CRISPR make expensive treatment accessible to all?