Gene therapy for genetic disorder affecting brain dopamine metabolism shows success; same delivery technique being investigated for PD

[Rhyothemis]

Really exciting report of disease reversal in children with AADC (aromatic L-amino acid decarboxylase deficiency); article mentions same delivery technique is being used for GDNF gene therapy in Parkinson's.

wexnermedical.osu.edu/depar...

[jimcaster]

Thanks for sharing. This is very encouraging.

“I’ve been investigating therapeutic gene therapy approaches for Parkinson’s disease for nearly 30 years, and this marks a significant milestone that may lead to major therapeutic opportunities,” says Dr. Bankiewicz, who founded the biotech startup Brain Neurotherapy Bio Inc. in 2018 to develop gene therapies for neurological disorders.

Bankiewicz and his team hope the Parkinson’s gene therapy surgery — a one-time treatment — provides ongoing benefits to slow disease progression.