Use of Gene Therapy for Dwarfism

Alexis Gorin
Farah Laiwalla
Engineering Biomedical Systems (BI920-3B)
Wednesday, July 30, 2008

To treat dwarfism, the condition of abnormally-caused small stature, doctors use gene therapy as an option to help people affected, using growth hormone and any other hormone the person may be missing. The amount of each type of hormone given constantly changes though due to (metabolic) changes within the child (1).

Different drug deliveries systems are used to get the growth hormone in the body systems. One way, used by doctors like Jeffery M. Leiden, is to remove muscle cells and place the gene that makes HGH inside the cells, only to be placed back inside the body (2). This method has become very effective since new research came out, proving that muscle cells can secrete proteins. The study shows that after three months, the mice the manipulated muscle cells were put in were still making HGH.

Another way to deliver the needed hormones, although can be used for other purposes, is to put the hormone-creating gene into "replication-deficient retrovirus" (3). The virus then can attach to muscle cells and has its host cell copy the hormone-creating genetic material, have the material build up, and then spread to other cells. This was created by Vandenburgh as a way to deal without constant injections of proteins.

(1) "Dwarfism." Free Health Encyclopedia. 2007. NetIndustries, LLC. . 30 Jul 2008 http://www.faqs.org/health/topics/99/Dwarfism.html.
(2) Angier, Natalie. "With Direct Injections, Gene Therapy Takes A Step Into a New Age ." The New York Times 14 April 1992 30 Jul 2008 http://query.nytimes.com/gst/fullpage.html?res=9E0CE6DB1E3BF937A25757C0A964958260&sec=&spon=&pagewanted=all.
(3) "Gene Therapy - Putting Muscle Into the Research." NASA. 06 June 2002. NASA. 30 Jul 2008 http://www.nasa.gov/vision/earth/livingthings/gene_therapy_prt.htm.