E-LIB Suche - Ergebnisse für: van Til, Niek P.

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/..

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

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/..

4

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/..

5

Advanced Therapy Medicinal Products: Clinical, Non-clinical..:

, In: Pharmaceutical Biotechnology,

Mastrobattista, Enrico ; Doevendans, Erik ; van Til, Niek P.... - p. 323-399 , 2024

Link: https://doi.org/10.1007/..

6

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/..

7

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/..

8

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/..

9

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/..

10

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/..

11

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/..

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

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/..

14

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/..

15

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/..