Modifying the activity of the gene that becomes mutated in Parkinson's disease may alter disease progression.
Scientists have found specific genetic mutations that appear to have a role in Parkinson’s disease. Mutations in the gene known as DJ-1 lead to the accelerated loss of neurons that produce dopamine, and this can lead to the early onset of Parkinson’s Disease symptoms such as shaking, rigidity, and difficulty walking.
Nirit Lev, PhD, MD, a researcher at Tel Aviv University’s Sackler Faculty of Medicine, and her team developed a peptide that mimics the normal function of DJ-1, a gene which protects the dopamine-producing neurons. By attaching the DJ-1-related peptide to a vehicle peptide, the researchers were able to deliver their engineered peptide through the cell membrane so it could be carried to the brain in mice models.
Results of the study showed that the peptide halted neurodegeneration, significantly reduced problems with movement and mobility, and created higher levels of dopamine in the brain. Based on these promising results, researchers think this method could be developed further as a preventative therapy for Parkinson’s disease well before the appearance of motor symptoms.
“Current treatments are lacking because they can only address symptoms—there is nothing that can change or halt the disease,” Dr. Lev said in a press release. “Until now, we have lacked tools for neuroprotection.”
The researchers noted that this type of peptide treatment has numerous benefits. It can be easily delivered by daily injections or absorbed into the skin via an adhesive patch. While most peptides degrade quickly, this particular formulation resists degradation for a longer period of time. Lastly, because peptides are organic to the body, this type of therapy would provide patients with a safer treatment option.