This company is developing lentiviral vectors (viruses) to deliver three genes that are involved in the production of dopamine.
So these viral vectors (viral particles) will be injected into the brain, introduce their genes into brain cells, and these genes will lead to the production of proteins that are involved in the production of dopamine.
However, with this approach the causes of Parkinson’s disease are not addressed. These causes (e.g. mitochondrial dysfunction, protein accumulation and others) lead to damage to the dopaminergic cells, leading to less dopamine being produced.
Furthermore, viral vectors can cause some damage (e.g. inflammation) when infecting cells. Given the dopaminergic cells in Parkinson’s patients are already damaged, and reside an an aged proinflammatory environment, this could not be a good thing.
So it remains to be seen if this approach could significantly slow the progression of the disease or reduce symptoms.
