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2025
Mark Gene therapy in advanced metachromatic leukodystrophy : tempering expectations
Schoenmakers, Daphne H. ; Beerepoot, Shanice ; Adang, Laura A. ; Asbreuk, Marije A.B.C. ; Bergner, Caroline G. ; Bley, Annette E. ; Boelens, Jaap Jan ; Calbi, Valeria ; Darling, Alejandra and Eklund, Erik ^LU , et al. (2025) In Protein and Cell 16(1). p.12-15
- Contribution to journal › Debate/Note/Editorial
Mark Metachromatic Leukodystrophy : New Therapy Advancements and Emerging Research Directions
Asbreuk, Marije A B C ; Schoenmakers, Daphne H ; Adang, Laura Ann ; Beerepoot, Shanice ; Bergner, Caroline ; Bley, Annette ; Boelens, Jaap Jan ; Bugiani, Marianna ; Calbi, Valeria and García-Cazorla, Àngeles , et al. (2025) In Neurology 105(2).
- Contribution to journal › Scientific review
2017
Mark Quality of life of Hurler syndrome patients after successful hematopoietic stem cell transplantation
Aldenhoven, Mieke ; Van Den Broek, Brigitte T.A. ; Wynn, Robert F. ; O'Meara, Anne ; Veys, Paul ; Rovelli, Attilio ; Jones, Simon A. ; Parini, Rossella ; Van Hasselt, Peter M. and Renard, Marleen , et al. (2017) In Blood Advances 1(24). p.2236-2242
- Contribution to journal › Article
2015
Mark Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation : An international multicenter study
Aldenhoven, Mieke ; Wynn, Robert F. ; Orchard, Paul J. ; O'Meara, Anne ; Veys, Paul ; Fischer, Alain ; Valayannopoulos, Vassili ; Neven, Benedicte ; Rovelli, Attilio and Prasad, Vinod K. , et al. (2015) In Blood 125(13). p.2164-2172
- Contribution to journal › Article
2007
Mark Lung delivery studies using siRNA conjugated to TAT(48-60) and penetratin reveal peptide induced reduction in gene expression and induction of innate immunity
Moschos, Sterghios Athanasios ; Jones, Simon Wyn ; Perry, Mark Michael ; Williams, Andrew Evan ; Erjefält, Jonas ^LU ; Turner, John James ; Barnes, Peter John ; Sproat, Brian Stephen ; Gait, Michael John and Lindsay, Mark Andrew (2007) In Bioconjugate Chemistry 18(5). p.1450-1459
- Contribution to journal › Article

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