Self Gene Therapy

Just read a story in MIT Technology Review. It is an attempt of self- experimentation on gene therapy by a microbiologist Brian Hanley. Hanley wanted to increase the level of Growth Hormone Releasing Hormone (GHRH) in human body by injecting the gene locally to muscles. He originally conceived it as a therapy for AIDS patients. GHRH go to pituitary to increase the Growth hormone, it also proposed to have an immunity-enhancing role that might help AIDS patients. But there were no takers for his idea. So he decided to employ himself as a test subject. With help of a local cosmetic surgeon he injected the plasmid (circular DNA) that encode the GHRH to his thigh muscles. Unlike the viral delivery mechanism he used electroporation, a method in which small electric shock given to cells to create small pores on cells membrane to facilitate intake of large molecules such as plasmids. Usually done in cell culture conditions. Apparently it has been performed in lab animals too for same purpose, but not in humans. Hanley’s blood was evaluated in George Church’s lab, a pioneer in genome engineering and synthetic biology in HMS. They found a slight elevation in GHRH level in Henley’s blood. That’s all known for now.

Questions: (i) Is it because of electroporation of plasmids (electroporation only control is not available).

(ii) What are the immediate benefits/side effects of GHRH over expression?

(iii) Does the over expression is maintained for a long period? How long? How? Was it taken up by some long surviving cells (stem cells)?

New information:

1. There are private Institutional Review Boards (IRB): IRB is a panel that determine the ethical aspect of the study if it involves a living subject. Hanley got the approval from the Institute of Regenerative and Cellular Medicine in Santa Monica, California, a private “institutional review board”.

2. Electroporation as a local in vivo DNA delivery strategy. How effective and targeted is it?

Apparently there is another self-experimentation story: Elizabeth Parrish, who voluntarily underwent a gene therapy (viral delivery) using genes for Follistatin (that inhibit myostatin, that decrease muscle growth), and telomerase, enzyme that regenerate telomeres, the DNA repeats that protect the end of the chromosome like and aglet protect a shoe lace.  Details of this experiment are also not known.