Niek Van Til

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In Vivo Base Editing of a Pathogenic Eif2b5 Variant Improves Vanishing White Matter Phenotypes in Mice

Desiree Bock, Ilma M. Revers, Anastasia S. J. Bomhof,Anne E. J. Hillen, Claire Boeijink,Lucas Kissling,Sabina Egli,Miguel A. Moreno-Mateos,Marjo S. van der Knaap,Niek P. van Til,Gerald Schwank

MOLECULAR THERAPYno. 5 (2024): 1328-1343

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Lentiviral Gene Therapy with IGF2-tagged GAA Normalizes the Skeletal Muscle Proteome in Murine Pompe Disease

Qiushi Liang,Joon M. Pijnenburg,Erikjan Rijkers,Arnold G. Vulto,Ans T. van der Ploeg,Niek P. van Til,Eva C. Vlaar,Jeroen A. A. Demmers,W. W. M. Pim Pijnappel

JOURNAL OF PROTEOMICS（2024）

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Combining CRISPR-Cas9 and TCR Exchange to Generate a Safe and Efficient Cord Blood-Derived T Cell Product for Pediatric Relapsed AML

Vania Lo Presti,Angelo Meringa,Ester Dunnebach, Alice van Velzen, Aida Valera Moreira,Ronald W. Stam,Rishi S. Kotecha,Anja Krippner-Heidenreich,Olaf T. Heidenreich,Maud Plantinga,Annelisa Cornel,Zsolt Sebestyen,Jurgen Kuball,Niek P. van Til,S. Nierkens

Journal for ImmunoTherapy of Cancerno. 4 (2024): e008174-e008174

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Human Post-Mortem Organotypic Brain Slice Cultures: a Tool to Study Pathomechanisms and Test Therapies

Bonnie C. Plug, Ilma M. Revers,Marjolein Breur, Gema Muñoz González,Jaap A. Timmerman, Niels R.C. Meijns, Daniek Hamberg, Jikke Wagendorp,Erik Nutma,Nicole I. Wolf,Antonio Luchicchi,Huibert D. Mansvelder,Niek P. van Til,Marjo S. van der Knaap,Marianna Bugiani

ACTA NEUROPATHOLOGICA COMMUNICATIONSno. 1 (2024)

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Assessment of Non-Myelotoxic Agents As a Preparatory Regimen for Hematopoietic Stem Cell Gene Therapy

Mehmet Emin Şeker,Özgür Doğuş Erol,Burcu Pervin,Gerard Wagemaker,Niek P. van Til,Fatima Aerts-Kaya

Human cellno. 1 (2024): 9-9

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P1372: NON-MYELOTOXIC CHEMICALS AS A PREPARATORY REGIMEN FOR HEMATOPOIETIC STEM CELL GENE THERAPY

Mehmet Emİn Şeker,Özgür Doğuş Erol,Burcu Pervin,Gerard Wagemaker,Niek P. van Til,Fatima Aerts‐Kaya

HemaSphereno. S3 (2023): e6316019-e6316019

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Focal Lesions Following Intracerebral Gene Therapy for Mucopolysaccharidosis IIIA

Marianna Bugiani,Truus E. M. Abbink,Arthur W. D. Edridge,Lia van der Hoek,Anne E. J. Hillen,Niek P. van Til,Gino V. Hu-A-Ng,Marjolein Breur,Karen Aiach,Philippe Drevot,Michael Hocquemiller,Ralph Laufer,Frits A. Wijburg,Marjo S. van der Knaap

Annals of Clinical and Translational Neurology（2023）

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Gene and Cellular Therapies for Leukodystrophies

Fatima Aerts-Kaya,Niek P. van Til

Pharmaceuticsno. 11 (2023): 2522

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Lentiviral Gene Therapy Prevents Anti-Human Acid Α-Glucosidase Antibody Formation in Murine Pompe Disease

Qiushi Liang,Eva C. Vlaar,Fabio Catalano,Joon M. Pijnenburg,Merel Stok,Yvette van Helsdingen,Arnold G. Vulto,Wendy W. J. Unger,Ans T. van der Ploeg,W. W. M. Pim Pijnappel,Niek P. van Til

Molecular therapy Methods & clinical development（2022）

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Long-Term Efficacy and Safety in Preclinical Hematopoietic Stem Cell Gene Therapy in Pompe Mice

Niek P. van Til,John K. Yoon,Rena Schindler,Yildirim Dogan,Cecilia Barese,Zeenath Unnisa,Mary Jacobs,Daniella Pizzurro,Christine Oborski,Michael Rothe,Axel Schambach,Aimin Yan,Luca Biasco,Richard Pfeifer,Chris Mason

MOLECULAR THERAPY（2022）

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论文共 75 篇作者统计合作学者相似作者