Pompe disease: towards gene correction using targeted nucleases
PhD ceremony: Ms. T.M. Geel, 14.45 uur, Academiegebouw, Broerstraat 5, Groningen
Thesis: Pompe disease: towards gene correction using targeted nucleases
Promotor(s): prof. M.G. Rots
Faculty: Medical Sciences
Pompe disease is an inherited metabolic disorder caused by a mutation in the gene encoding the enzyme acid-α-glucosidase (GAA). In this dissertation, novel strategies were investigated to improve the treatment of Pompe disease. The only currently available therapy (Enzyme Replacement Therapy) is hampered by several disadvantages such as inefficient uptake of the enzyme. We demonstrated that a known efficient DNA carrier (SAINT) could also be used to effectively deliver proteins, including lysosomal enzymes and restriction enzymes.
| Last modified: | 13 March 2020 01.16 a.m. |
More news
-
27 August 2024
UMCG gaat onderzoeksfaciliteiten beschikbaar stellen voor geneesmiddelenontwikkeling
Om de beschikbaarheid en effectiviteit van geneesmiddelen in Nederland te verbeteren gaat het UMCG het bedrijf G² Solutions opzetten. Dit bedrijf moet ervoor gaan zorgen dat belangrijke technologische ontwikkelingen op het gebied van DNA sequencing...
-
17 July 2024
Veni-grants for ten researchers
The Dutch Research Council (NWO) has awarded a Veni grant of up to €320,000 each to ten researchers of the University of Groningen and the UMCG. The Veni grants are designed for outstanding researchers who have recently gained a PhD.
-
16 July 2024
Medicine still subjects to male bias
Aranka Ballering studied the course of illness in people with common symptoms. One of the most striking findings to emerge from her research was that on average, women have a different – and less extensive – course of illness than men.