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
-
13 May 2024
Trapping molecules
In his laboratory, physicist Steven Hoekstra is building an experimental set-up made of two parts: one that produces barium fluoride molecules, and a second part that traps the molecules and brings them to an almost complete standstill so they can...
-
06 May 2024
Impact: 'URinControl', treatment of urine loss through app
One of the three researchers nominated for the Ben Feringa Impact Award 2024 is general practicioner and researcher-epidemiologist Anne Loohuis. Loohuis conducted research on the treatment of urine loss through the self-help app URinControl'.
-
29 April 2024
Royal Decoration awarded to Gerard van den Berg
The Faculty of Economics and Business is very proud to announce that Professor Gerard van den Berg has been appointed Officer of the Order of Orange-Nassau. The decoration was presented to him by Mayor Koen Schuiling of the Municipality of...