Validating a clinical candidate lentiviral vector for human haematopoietic stem cell gene therapy trials in people with Friedreich’s ataxia

Friedreich’s Ataxia (FA) is a complex and currently incurable genetic disorder, that typically appears in late childhood. People with the condition experience progressive accumulation of neurological disability. The disorder also affects multiple other organs, with over half of FA patients also presenting with cardiomyopathy, and a third with…