Dr Joanne Ng
Our long term aim is to develop novel translational therapies for genetic paediatric neurological disorders that are intractable or pharmacoresistant. We work on a range of disorders including childhood movement disorders namely dystonia-parkinsonism and childhood epilepsies. In parallel we aim to develop novel platforms to screen viral vectors with selective expression to improve expression efficiency and viral gene therapy safety.
Paediatric Neurotransmitter and Juvenile Parkinsonism
In collaboration with Professors Manju Kurian and Simon Waddington we developed a translational gene therapy for Dopamine transporter deficiency syndrome (DTDS) a paediatric neurotransmitter disorder that is progressing towards clinical trial. The research group are developing gene therapies other paediatric neurotransmitter disorders and juvenile parkinsonism.
Novel CNS promoters
With Riccardo Privolizzi and AskBio Europe, this collaboration is developing novel synthetic selective gene therapy promoters for CNS application and new platforms to increase screening throughput.