A particular antibiotic mutated by researchers at the University of Florida has represented the first step in the curing of Parkinson’s disease. An article that was published in the September issue of Molecular Therapy has shown that a particular antibiotic that was mainly used to try and cure Parkinson’s disease can also function as a cure for gene therapy. The study which was conducted in rats now shows how therapeutic genes which when delivered to the human brain can help in the treatment of Parkinson's disease.
adeno associated virus (source Wikipedia)
Says Ronald Mandel, a professor of neuroscience at the Powell Gene Therapy Center and University of Florida‘s McKnight Brain Institute, “Most prior experiments that made use of any growth factors that grew naturally in the body to divide and grow cells as a means of rejuvenating dying brain cells failed due to the fact that they were injected too late. However, newer findings suggest that it is possible for the brain to produce a neurotransmitter known as dopamine mainly thanks to gene therapy.” Patients in Parkinson’s disease often face a shortfall in the production of dopamine, but now due to the latest findings of the research, there would be an increased likelihood of success. Dopamine is very essential as it helps in the communication and coordination between cells.
“We have spent over 10 years trying to design a gene delivery vector that can help in safely transferring the genes required for Parkinson’s. We now believe that it is possible to intervene at the earliest stages of the disease as some of our earlier interventions failed to save dopamine-producing connections in patients mainly due to the fact that they were given to suffering patients only during the latter stages of the disease and as such there were very few dopamine-producing connections left,” says Mandel.
The Mandel led team of scientists used an adeno-associated virus that was capable of inducing dopamine cells into animal brain cells which could then result in the production of GDNF. This GDNF is vital as it helps the dopamine-producing neurons survive during the process of development of the brain of adults.
The virus was engineered by scientists with two genes so as to be able to act in connotation with each other to produce protein. By using an antibiotic called dietary doxycycline, the progression of the genes could be slowed down. The amount of the antibiotic determines the regulation of production of the protein. In other words, it is even possible to reduce this protein which in turn can give medical investigators the option of regulating gene therapy after delivery of the treatment.


