A naturally myostatin blocked boy. Antisense RNA has been used to activate myostatin inhibition in mice
Mice lacking the myostatin gene have 25–30% increased muscle mass. Individual muscles, such as the pectoralis and quadriceps, of myostatin mutant mice are two- to threefold heavier than those of wild type mice.
Antisense RNAs were injected into normal mice through intravenous (i.v.) routes at the dose of 5 mg kg-1, twice weekly for 4 weeks. Two days after the last injection, the leg muscles were weighed and the ratio of the leg’s muscle weight to body weight in each group was calculated
So it is a good thing that they have found that gene therapy can be used to increase tendon strength in rats by 70%.
Tendons transduced with BMP-14 exhibited less visible gapping, a greater number of neotenocytes at the site of healing, and 70% greater tensile strength than did either those transduced with GFP or the sham controls at two weeks after repair. Histological examination revealed no inflammatory response to the adenovirus in tendons transduced with BMP-14 or GFP. No ectopic bone or cartilage formed in the tendons transduced with BMP-14.
They believe this will help tendons heal.
Gene therapy could one day help cure diseases like cancer, aids and diabetes and it’s been estimated that by 2011, the market for gene therapy products will be US$6.5 billion.
The new method approaches treatment of the AIDS virus by using modified HIV virus, called VRX496, to attack and destroy the protective shell, or envelope, of the virus within living cells.
Typically, viruses reproduce by taking over the replication machinery of a living cell, turning the cell into a factory that pumps out more copies of the virus.
By attacking the protective envelope of the HIV virus, the new therapy suppresses the viability of the HIV virus, meaning that the virus can no longer replicate itself as successfully.
However, the possible changes to the human body don’t end with disease.Researchers have also been able to reduce fat, pump up muscle (myostatin blockers), prevent and help recovery from radiation damage, lengthen lifespans and change the color of mice through gene manipulation. Although gene therapy is not yet considered safe for humans, it is only a matter of time before better techniques arrive that make it possible on a large scale.
Gene therapy is leading to faster and cheaper drug development and production.
It is quicker to make transgenic animals using gene therapy. Current methods of producing such animals involves microinjection and cloning, which is a more expensive and longer process. These methods are inefficient and also carry a risk of producing offspring with developmental abnormalities. Instead of 100 to 300 attempts to make clones that work, 10% of animals were breeding the desired changes with gene therapy.
RNA interference (RNAi) for gene therapy
At least six clinical trials using RNA interference (RNAi) have been approved, “with many more coming down the pipeline,” according to the Editorial by Mark A. Kay, MD, PhD, an Associate Editor of Human Gene Therapy and the Dennis Farrey Family Professor in Pediatrics and Professor of Genetics at Stanford University School of Medicine. “One thing is clear,” adds Kay, “small RNAs as a therapeutic platform are here to stay.”
The rats were injected with a gene that tricks the body into releasing endorphins, a natural painkiller, in the nerve cells surrounding the spinal cord.
The treatment simulates the effect of painkilling drugs but is much narrower in scope, targeting nerve cells along the spinal cord, but not in the brain or in other parts of the central nervous system.
Myostatin inhibitor trials on humans