Dr. Haiyan Fu of The Research Institute at Nationwide Children's Hospital, Ohio State University, Colombus, OH, recently presented an update on her research at the 11th International Symposium on Mucopolysaccharide and Related Diseases in Adelaide, Australia.

Dr. Fu and her team believe the results justify moving her gene therapy approach to Human Clinical Trial. The final slide from her presentation at Adelaide provides the best summary:

  • A single IV injection of rAAV9-hNaGlu vector injection resulted in widespread expression of rNaGlu, the correction of lysosomal storage pathology in the CNS and somatic tissues, and the correction of secondary CNS pathology.
  • The treatment led to functional correction of the neurological disease.
  • Much lower dose of rAAV9 vector is required to achieve significant functional benefits, compared to rAAV2 that eases the challenge in the mouse-humans translation of gene therapy, scalable vector production.
Trans-BBB neurotropism and minimal possibility of pre-existing immunity in humans: rAAV9 is the vector to go for clinical application in patients with MPS IIIB and other LSD with neurological involvement.

Research Summaries