Exploiting anti-capsid humoral immunity induced in infants receiving gene therapy for Spinal Muscular Atrophy to engineer the next generation of gene transfer vectors [ 1/1/2021 - 12/31/2023 ]

Research Grant

Brief description After 25 years of incremental progress the possibility of treating genetic disease by gene therapy has become a therapeutic reality. This has been achieved by harnessing the gene transfer power of viruses made harmless by genetic engineering. A major limitation is that up to 50% of patients are currently excluded by pre-existing immunity to these powerful tools. Using 'evolution in a dish', we will engineer a new generation of these tools capable of bypassing pre-existing immunity by stealth.

Funding Amount $1,105,993

Funding Scheme Ideas Grants

Chief Investigator Prof Ian Alexander

Notes Ideas Grants