Specialization
Adeno associated virus gene therapy, preclinical studies, efficacy and safety testing, hematopoietic stem cell gene therapy, genome editing, metabolic diseases and leukodystrophies.
Focus of research
Niek van Til has over 20 years of experience in the gene therapy field, currently focussing on preclinical development of tailor-made gene and cell therapies for leukodystrophies at the Amsterdam Leukodystrophy Center. The pipelines include the development of adeno-associated virus (AAV) vectors to deliver genetic therapeutics to the central nervous system for treatment of the leukodystrophy vanishing white matter. In addition, ex vivo hematopoietic stem cell gene therapy using lentiviral vectors are investigated for other leukodystrophies in which cross-correction is a mechanism of action. Other groundbreaking technologies, such as precise genome editing are also explored to treat these debilitating genetic diseases for which there is currently no cure.