This company is developing AAVs (viral vectors) which encode for antibodies and also contain miRNA to suppress the production of alpha-synuclein protein in brain cells.
Alpha-synuclein is a protein that accumulates in Parkinson’s disease.
This therapy focuses on people with mutations in the SNCA gene, which is the gene that encodes the alpha-synuclein protein.
However, it remains to be seen if this therapy will work. Alpha-synuclein is a protein that performs many tasks in the brain, ranging from forming little vesicles in cells to DNA repair - even mutated alpha-synuclein could still (partly) perform such tasks.
Reducing the amounts of this important brain protein could lead to significant side effects.
