CRISPR/Cas9-Mediated Gene Editing of Haematopoietic Stem and Progenitor Cells for Friedreich’s Ataxia

Friedreich’s ataxia (FRDA) is an autosomal recessive disease caused by loss-of-function mutations in the FXN gene, which encodes the mitochondrial protein frataxin. Frataxin plays roles in several key processes in iron metabolism, antioxidant protection, and energy production in the mitochondria.

With no effective treatments, patients with this condition experience progressive loss of motor skills and degeneration of muscle tissue and sensory nerve fibres throughout the body, eventually resulting in the inability to walk. The majority of FRDA patients die before the age of 40.

Professor Stephanie Cherqui of University of California San Diego’s School of Medicine recently received a prestigious grant from the Californian Institute of Regenerative Medicine, which will allow her and her team to continue their groundbreaking research on FRDA and bring a novel CRISPR therapy to the clinic.

What will you learn from this webinar:

• Impact of haematopoietic stem cell transplantation for Friedreich’s ataxia

• Optimisation of the CRISPR/Cas9-mediated gene editing approach of the patients' haematopoietic stem cells

• Mechanism of neuronal rescue after haematopoietic stem cell transplantation in Friedreich’s ataxia

Webinar Programme:

• 15.00 Welcome and introduction by CRISPR Medicine News
• 15.05 Professor Stephanie Cherqui |CRISPR/Cas9-Mediated Gene Editing of Haematopoietic Stem and Progenitor Cells for Friedreich’s Ataxia
• 15.45 Q & A with Professor Stephanie Cherqui
• 16.00 Close by CRISPR Medicine News

Speaker | Title:

Professor Stephanie Cherqui, University of California, San Diego | CRISPR/Cas9-Mediated Gene Editing of Haematopoietic Stem and Progenitor Cells for Friedreich’s Ataxia