Research output per year
Research output per year
- 5 results
Search results
-
2024
Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage
Catalano, F., Vlaar, E. C., Dammou, Z., Katsavelis, D., Huizer, T. F., Zundo, G., Hoogeveen-Westerveld, M., Oussoren, E., van den Hout, H. J. M. P., Schaaf, G., Pike-Overzet, K., Staal, F. J. T., van der Ploeg, A. T. & Pijnappel, W. W. M. P., 1 Apr 2024, In: Human Gene Therapy. 35, 7-8, p. 256-268 13 p.Research output: Contribution to journal › Article › Academic › peer-review
Open AccessFile22 Downloads (Pure) -
2023
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II
Catalano, F., Vlaar, E. C., Katsavelis, D., Dammou, Z., Huizer, T. F., van den Bosch, J. C., Hoogeveen-Westerveld, M., van den Hout, H. J. M. P., Oussoren, E., Ruijter, G. J. G., Schaaf, G., Pike-Overzet, K., Staal, F. J. T., van der Ploeg, A. T. & Pijnappel, W. W. M. P., 14 Dec 2023, In: Molecular Therapy Methods and Clinical Development. 31, 101149.Research output: Contribution to journal › Article › Academic › peer-review
Open AccessFile3 Citations (Scopus)17 Downloads (Pure) -
2022
IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy
Liang, Q., Catalano, F., Vlaar, E. C., Pijnenburg, J. M., Stok, M., van Helsdingen, Y., Vulto, A. G., van der Ploeg, A. T., van Til, N. P. & Pijnappel, W. W. M. P., 8 Dec 2022, In: Molecular Therapy - Methods and Clinical Development. 27, p. 109-130 22 p.Research output: Contribution to journal › Article › Academic › peer-review
Open AccessFile19 Citations (Scopus)79 Downloads (Pure) -
Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease
Liang, Q., Vlaar, E. C., Catalano, F., Pijnenburg, J. M., Stok, M., van Helsdingen, Y., Vulto, A. G., Unger, W. W. J., van der Ploeg, A. T., Pijnappel, W. W. M. P. & van Til, N. P., 9 Jun 2022, In: Molecular Therapy - Methods and Clinical Development. 25, p. 520-532 13 p.Research output: Contribution to journal › Article › Academic › peer-review
Open AccessFile15 Citations (Scopus)54 Downloads (Pure) -
2021
A generic assay for the identification of splicing variants that induce nonsense-mediated decay in Pompe disease
Bergsma, A. J., in ’t Groen, S. L. M., Catalano, F., Yamanaka, M., Takahashi, S., Okumiya, T., van der Ploeg, A. T. & Pijnappel, W. W. M. P., Mar 2021, In: European Journal of Human Genetics. 29, 3, p. 422-433 12 p.Research output: Contribution to journal › Article › Academic › peer-review
Open AccessFile7 Citations (Scopus)30 Downloads (Pure)