Dr. Niek van Til

Niek van Til, a medical biologist, has worked on gene therapy for immune deficiencies, metabolic disorders and cellular immunotherapy for pediatric cancer.

At the ALC, he focusses on preclinical efficacy and safety assessment of gene therapies for leukodystrophies.

Friday May 23 2024, a day on therapy development in leukodystrophies 9.00 – 9.30      Envisioning the future of gene therapy – Niek van Til

In the last two decades, gene therapy has become a reality for treating patients with leukodystrophies. These genetic technologies are developing fast and are transforming the landscape of available treatment options. Approaches such as gene augmentation using viral delivery systems and more recently developed groundbreaking genome editing approaches, open up opportunities to directly address correction at the root cause of these genetic diseases. Alternatively, ex vivo hematopoietic stem cell gene therapy has become a treatment modality for selected leukodystrophies, in which microglia are affected or if cross-correction is the mechanism-of-action for correction. The clinical spectrum of these leukodystrophies is diverse and tailor-made therapies are required for successful future treatment applications.