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van Til, Niek P.
355
results:
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1
Combining CRISPR-Cas9 and TCR exchange to generate a safe a..:
Lo Presti, Vania
;
Meringa, Angelo
;
Dunnebach, Ester
...
Journal for ImmunoTherapy of Cancer. 12 (2024) 4 - p. e008174 , 2024
Link:
https://doi.org/10.1136/..
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2
In vivo base editing of a pathogenic Eif2b5 variant improve..:
Böck, Desirée
;
Revers, Ilma M.
;
Bomhof, Anastasia S.J.
...
Molecular Therapy. 32 (2024) 5 - p. 1328-1343 , 2024
Link:
https://doi.org/10.1016/..
?
3
Lentiviral gene therapy with IGF2-tagged GAA normalizes the..:
Liang, Qiushi
;
Vlaar, Eva C.
;
Pijnenburg, Joon M.
...
Journal of Proteomics. 291 (2024) - p. 105037 , 2024
Link:
https://doi.org/10.1016/..
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4
Human post-mortem organotypic brain slice cultures: a tool ..:
Plug, Bonnie C.
;
Revers, Ilma M.
;
Breur, Marjolein
...
Acta Neuropathologica Communications. 12 (2024) 1 - p. , 2024
Link:
https://doi.org/10.1186/..
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5
Focal lesions following intracerebral gene therapy for muco..:
Bugiani, Marianna
;
Abbink, Truus E. M.
;
Edridge, Arthur W. D.
...
Annals of Clinical and Translational Neurology. 10 (2023) 6 - p. 904-917 , 2023
Link:
https://doi.org/10.1002/..
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6
Gene and Cellular Therapies for Leukodystrophies:
Aerts-Kaya, Fatima
;
van Til, Niek P.
Pharmaceutics. 15 (2023) 11 - p. 2522 , 2023
Link:
https://doi.org/10.3390/..
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7
Gene Therapy Developments for Pompe Disease:
Unnisa, Zeenath
;
Yoon, John K.
;
Schindler, Jeffrey W.
..
Biomedicines. 10 (2022) 2 - p. 302 , 2022
Link:
https://doi.org/10.3390/..
?
8
High-throughput analysis of hematopoietic stem cell engraft..:
Plasschaert, Robert N.
;
DeAndrade, Mark P.
;
Hull, Fritz
...
Molecular Therapy. 30 (2022) 10 - p. 3209-3225 , 2022
Link:
https://doi.org/10.1016/..
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9
Screening chimeric GAA variants in preclinical study result..:
Dogan, Yildirim
;
Barese, Cecilia N.
;
Schindler, Jeffrey W.
...
Molecular Therapy - Methods & Clinical Development. 27 (2022) - p. 464-487 , 2022
Link:
https://doi.org/10.1016/..
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10
Long-term hematopoietic stem cell lentiviral gene therapy r..:
van Til, Niek P.
;
Schindler, Jeffrey W.
;
Yoon, John K.
...
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S122-S123 , 2022
Link:
https://doi.org/10.1016/..
?
11
Lentiviral gene therapy prevents anti-human acid α-glucosid..:
Liang, Qiushi
;
Vlaar, Eva C.
;
Catalano, Fabio
...
Molecular Therapy - Methods & Clinical Development. 25 (2022) - p. 520-532 , 2022
Link:
https://doi.org/10.1016/..
?
12
IGF2-tagging of GAA promotes full correction of murine Pomp..:
Liang, Qiushi
;
Catalano, Fabio
;
Vlaar, Eva C.
...
Molecular Therapy - Methods & Clinical Development. 27 (2022) - p. 109-130 , 2022
Link:
https://doi.org/10.1016/..
?
13
Long-term hematopoietic stem cell gene therapy corrects neu..:
van Til, Niek P.
;
Dogan, Yildirim
;
Barese, Cecilia
...
Molecular Genetics and Metabolism. 132 (2021) 2 - p. S107 , 2021
Link:
https://doi.org/10.1016/..
?
14
Efficient lentiviral transduction method to gene modify cor..:
Lo Presti, Vania
;
Cornel, Annelisa M.
;
Plantinga, Maud
...
Molecular Therapy - Methods & Clinical Development. 21 (2021) - p. 357-368 , 2021
Link:
https://doi.org/10.1016/..
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15
Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Mu..:
Stok, Merel
;
de Boer, Helen
;
Huston, Marshall W.
...
Molecular Therapy - Methods & Clinical Development. 17 (2020) - p. 1014-1025 , 2020
Link:
https://doi.org/10.1016/..
1-15