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RO
SETU gene therapy researcher inspired by family to tackle rare disease
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Silicon Republic
The gene therapy research that we are carrying out now is focused on developing an enhanced gene therapy for a particular rare neurodegenerative disorder called Canavan disease.
These internationally recognised gene therapy researchers had already developed Adeno-associated viral (AAV) delivery vectors and had gene therapy solutions under study, but I wanted to apply my experience of gene evolution and enzyme enhancement to contribute if possible.
Canavan disease is caused by accumulation of N-acetyl aspartate (NAA) in the brain, due to the deficiency of aspartoacylase (ASPA) enzyme, associated with mutations in the ASPA gene.
Gene therapy solutions being developed focus on delivering the ASPA gene past the blood-brain barrier and therefore enabling healthy ASPA enzyme production.
If an enhanced gene therapy reaches the market, a single dose should give more efficacy or benefit to the patient.
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