One-time treatment generates new neurons, eliminates Parkinson's disease in mice

What is it about PTB that makes this work? "This protein is present in a lot of cells," Fu said. "But as neurons begin to develop from their precursors, it naturally disappears. What we've found is that forcing PTB to go away is the only signal a cell needs to turn on the genes needed to produce a neuron."

Of course, mice aren't people, he cautioned. The model the team used doesn't perfectly recapitulate all essential features of Parkinson's disease. But the study provides a proof of concept, Fu said.

Next, the team plans to optimize their methods and test the approach in mouse models that mimic Parkinson's disease through genetic changes. They have also patented the PTB antisense oligonucleotide treatment in order to move forward toward testing in humans.

"It's my dream to see this through to clinical trials, to test this approach as a treatment for Parkinson's disease, but also many other diseases where neurons are lost, such as Alzheimer's and Huntington's diseases and stroke," Fu said. "And dreaming even bigger—what if we could target PTB to correct defects in other parts of the brain, to treat things like inherited brain defects?

https://medicalxpress.com/news/2020-06-one-time-treatment-neurons-parkinson-disease.html