Spencer Knight: Breakthrough in Gene Therapy – Life-Changing Vision Restored for Young Girl
Spencer Knight, Business Development Director at Hartmann Young, shared a post on LinkedIn:
“Breakthrough in Gene Therapy: Life-Changing Vision Restored for Young Girl
Saffie Sandford, a six-year-old girl from Stevenage, was diagnosed with a rare inherited eye condition called Leber’s Congenital Amaurosis (LCA) – a disorder that can lead to complete blindness.
Without treatment, her family were told she could lose her sight by adulthood.
In a groundbreaking procedure at Great Ormond Street Hospital, doctors used a one-off gene therapy called Luxturna, injecting a healthy copy of the faulty gene directly into her eyes.
The results have been remarkable.
After struggling to see in low light for years, Saffie can now see in the dark something her family described as ‘like a magic wand.’
She’s now doing everyday things many take for granted, from playing outside at night to enjoying activities she once had to miss out on.
The treatment has significantly improved her vision and quality of life, especially because it was given at such a young age and highlights the power of gene therapy to transform lives of children with rare genetic conditions.
Incredible to see science making the impossible possible.”
Stay updated with Hemostasis Today.
-
Jun 15, 2026, 15:36Alejandro González Veliz: A Coronary Calcium Score of Zero Doesn’t Always Mean Zero Risk
-
Jun 15, 2026, 15:35Tareq Abadl: Your Blood Tells Your Story – Even in the Shape of Its Cells
-
Jun 15, 2026, 15:34Three Days of Learning, Collaboration, and Community at EHC 2026
-
Jun 15, 2026, 15:31Abdul Mannan: What if Our First Move in Acquired Haemophilia A is Costing Frail Patients Time?
-
Jun 15, 2026, 14:11Alba Maiques Diaz: Completion of My Tenure in the YoungEHA Committee at the EHA
-
Jun 15, 2026, 13:51Miguel Gómez Álvarez: Navigating the Complexities of Erythrocytosis and the Role of PIEZO1
-
Jun 15, 2026, 13:37Katja Sockel: EHA 2026 Inspires New Ideas and Global Connections
-
Jun 15, 2026, 13:25Srishti Goyal։ India’s Blood Donation Journey – Looking Beyond the Numbers
-
Jun 15, 2026, 13:22Debra BenAvram: Every Two Seconds – Someone Needs Blood