Gene therapy treatment for ALS Gene therapy approach for C9ORF72 linked ALS: using AAV9 as a vehicle to deliver an artificial microRNA to target aberrant transcripts. A single dose will permanently treat motor neurons and prevent cell death. | Sci Ani

161K views · 1.3K reactions | A 5-year-old boy who was given a 'death sentence' due to a rare and severe immune disease is now living a normal life, thanks to groundbreaking gene therapy. Eisa's dad, Safdar and Professor Claire Booth, Head of Infection, Immunity and Inflammation Department at Great Ormond Street, discuss the life-changing treatment. | Good Morning Britain | Facebook