AAV-mediated therapy for visual impairment associated with Friedreich’s Ataxia
Evaluation of ARMMs-mediated delivery of Cas9 protein complexed with gRNAs as a non-viral disease-modifying strategy for Friedreich’s Ataxia
Engineering adeno-associated viral vectors to evade immune responses
Therapeutic activity of a haematopoietic stem cell delivered tissue penetrating peptide in a Friedreich’s ataxia mouse model
Regulation of frataxin expression – implications for Friedreich’s ataxia therapy
Defining the therapeutic window and threshold for neuronal gene therapy in Friedreich Ataxia
Development of autologous transplantation of genetically corrected hematopoietic stem cells for Friedreich Ataxia