Genetic Modification to prevent Parkinson’s Disease
Let me share with you one of the greatest breakthroughs in medical science in recent months, especially as this is with regard to a disease which I cover in my new book, ‘The Making of a Superhuman’.
Parkinson’s Disease, as many of you might know, is a debilitating neurological disease that affects brain cells and causes dopamine levels to drop and cause tremors, difficulty in movements, slowness and loss of balance. While the disease can be managed in the short-term by medications that boost dopamine, it is a progressive condition that worsens over time, and no cure has been discovered yet.
This is all set to change as a team of scientists at University of California San Diego School of Medicine, led by Xiang-Dong Fu, PhD, has discovered a breakthrough method to grow dopamine producing neurons, which could lead to a one-time treatment for eliminating Parkinson’s Disease from a patient’s body
Until now, growing dopamine producing neurons were thought to be next to impossible. While many research teams were trying to achieve it from stem cells, so far success had eluded all of them. But now this US team has achieved it through another novel way, which was part serendipity, part innovation.
The first breakthrough came when Fu and one of his team members accidentally discovered that when a gene called PTB was inhibited in connective tissue cells called fibroblasts, most of them were converted into neurons! Stunned at their discovery, Fu and another team member, Hao Qian, PhD, tried the same technique of inhibiting the PTB gene in another kind of cells called astrocytes that are abundant in brain. And voila, around 30% of these cells became dopamine producing neurons!
This study was done in brains of mice and it was made possible due to the presence of another heavyweight researcher in the team, Don Cleveland, PhD, who had earlier pioneered ‘Designer DNA Drugs’ which are technically artificial pieces of DNA called Antisense Oligonucleotide Sequences. The San Diego team developed a noninfectious virus that carried such a DNA sequence designed to inhibit the PTB gene, and thus enable astrocytes to be converted into neurons. And this worked perfectly, with the symptoms of Parkinson’s Disease getting eliminated from the treated mice!
While much more mouse studies and human studies remain to be done before this will progress to be a treatment, I think this technique of inhibiting a single gene to produce such remarkable results against a progressive disease like Parkinson’s holds immense promise in developing such one-shot cures against other such diseases and even many congenital conditions.