PTC is excited to announce the publication of “Gene therapy in the putamen for curing AADC deficiency and Parkinson’s disease” in the EMBO Molecular Medicine journal online with open access.
Featuring collaboration from medical experts Paul Wuh-Liang Hwu, Karl Kiening, lrina Anselm, David R. Compton, Takeshi Nakajima, Thomas Opladen, Phillip L. Pearl, Agathe Roubertie, Thomas Roujeau, and Shin-ichi Muramatsu – this study provides an overview of the putamen as an established target site for gene therapy in treating aromatic l-amino acid decarboxylase (AADC) deficiency and Parkinson’s disease – both rare disorders with high unmet medical need.
These two debilitating neurological disorders involve motor dysfunction caused by dopamine deficiencies. Learn more about how gene therapy can be delivered by using low doses of adeno-associated viral vectors to this acutely-defined area of the human brain.
This information is intended for health care professionals. This EMBO article contains product information.