Gene and Cellular Therapies for Leukodystrophies

por Aerts-Kaya, Fatima, van Til, Niek P.
Publicado 2023

Gene Augmentation and Editing to Improve TCR Engineered T Cell Therapy against Solid Tumors

por Lo Presti, Vania, Buitenwerf, Frank, van Til, Niek P., Nierkens, Stefan
Publicado 2020

Strategies to Genetically Modulate Dendritic Cells to Potentiate Anti-Tumor Responses in Hematologic Malignancies

por Cornel, Annelisa M., van Til, Niek P., Boelens, Jaap Jan, Nierkens, Stefan
Publicado 2018

Gene Therapy Developments for Pompe Disease

por Unnisa, Zeenath, Yoon, John K., Schindler, Jeffrey W., Mason, Chris, van Til, Niek P.
Publicado 2022

Reduction of liver macrophage transduction by pseudotyping lentiviral vectors with a fusion envelope from Autographa californica GP64 and Sendai virus F2 domain

por Markusic, David M, van Til, Niek P, Hiralall, Johan K, Elferink, Ronald PJ Oude, Seppen, Jurgen
Publicado 2009

Mitochondrial Neurogastrointestinal Encephalomyopathy Caused by Thymidine Phosphorylase Enzyme Deficiency: From Pathogenesis to Emerging Therapeutic Options

por Yadak, Rana, Sillevis Smitt, Peter, van Gisbergen, Marike W., van Til, Niek P., de Coo, Irenaeus F. M.
Publicado 2017

A “No‐Touch” Antibody‐Staining Method of Adherent Cells for High‐Throughput Flow Cytometry in 384‐Well Microplate Format for Cell‐Based Drug Library Screening

por Cornel, Annelisa M., Szanto, Celina L., van Til, Niek P., van Velzen, Jeroen F., Boelens, Jaap J., Nierkens, Stefan
Publicado 2019

Efficient lentiviral transduction method to gene modify cord blood CD8(+) T cells for cancer therapy applications

por Lo Presti, Vania, Cornel, Annelisa M., Plantinga, Maud, Dünnebach, Ester, Kuball, Jurgen, Boelens, Jaap Jan, Nierkens, Stefan, van Til, Niek P.
Publicado 2021

Transplantation, gene therapy and intestinal pathology in MNGIE patients and mice

por Yadak, Rana, Boot, Max V., van Til, Niek P., Cazals-Hatem, Dominique, Finkenstedt, Armin, Bogaerts, Elly, de Coo, Irenaeus F., Bugiani, Marianna
Publicado 2018

Alteration of viral lipid composition by expression of the phospholipid floppase ABCB4 reduces HIV vector infectivity

por van Til, Niek P, Heutinck, Kirstin M, van der Rijt, Roos, Paulusma, Coen C, van Wijland, Michel, Markusic, David M, Elferink, Ronald PJ Oude, Seppen, Jurgen
Publicado 2008

Angiopoietin-Like Protein 3 Promotes Preservation of Stemness during Ex Vivo Expansion of Murine Hematopoietic Stem Cells

por Farahbakhshian, Elnaz, Verstegen, Monique M., Visser, Trudi P., Kheradmandkia, Sima, Geerts, Dirk, Arshad, Shazia, Riaz, Noveen, Grosveld, Frank, van Til, Niek P., Meijerink, Jules P. P.
Publicado 2014

Physiological tonicity improves human chondrogenic marker expression through nuclear factor of activated T-cells 5 in vitro

por van der Windt, Anna E, Haak, Esther, Das, Ruud HJ, Kops, Nicole, Welting, Tim JM, Caron, Marjolein MJ, van Til, Niek P, Verhaar, Jan AN, Weinans, Harrie, Jahr, Holger
Publicado 2010

Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors

por Alonso-Ferrero, Maria E., van Til, Niek P., Bartolovic, Kerol, Mata, Márcia F., Wagemaker, Gerard, Moulding, Dale, Williams, David A., Kinnon, Christine, Waddington, Simon N., Milsom, Michael D., Howe, Steven J.
Publicado 2018

IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy

por Liang, Qiushi, Catalano, Fabio, Vlaar, Eva C., Pijnenburg, Joon M., Stok, Merel, van Helsdingen, Yvette, Vulto, Arnold G., van der Ploeg, Ans T., van Til, Niek P., Pijnappel, W.W.M. Pim
Publicado 2022

Novel immortalized human fetal liver cell line, cBAL111, has the potential to differentiate into functional hepatocytes

por Deurholt, Tanja, van Til, Niek P, Chhatta, Aniska A, ten Bloemendaal, Lysbeth, Schwartlander, Ruth, Payne, Catherine, Plevris, John N, Sauer, Igor M, Chamuleau, Robert AFM, Elferink, Ronald PJ Oude, Seppen, Jurgen, Hoekstra, Ruurdtje
Publicado 2009

Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease

por Stok, Merel, de Boer, Helen, Huston, Marshall W., Jacobs, Edwin H., Roovers, Onno, Visser, Trudi P., Jahr, Holger, Duncker, Dirk J., van Deel, Elza D., Reuser, Arnold J.J., van Til, Niek P., Wagemaker, Gerard
Publicado 2020

Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease

por Liang, Qiushi, Vlaar, Eva C., Catalano, Fabio, Pijnenburg, Joon M., Stok, Merel, van Helsdingen, Yvette, Vulto, Arnold G., Unger, Wendy W.J., van der Ploeg, Ans T., Pijnappel, W.W.M. Pim, van Til, Niek P.
Publicado 2022

Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease

por Dogan, Yildirim, Barese, Cecilia N., Schindler, Jeffrey W., Yoon, John K., Unnisa, Zeenath, Guda, Swaroopa, Jacobs, Mary E., Oborski, Christine, Maiwald, Tim, Clarke, Diana L., Schambach, Axel, Pfeifer, Richard, Harper, Claudia, Mason, Chris, van Til, Niek P.
Publicado 2022

High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing

por Plasschaert, Robert N., DeAndrade, Mark P., Hull, Fritz, Nguyen, Quoc, Peterson, Tara, Yan, Aimin, Loperfido, Mariana, Baricordi, Cristina, Barbarossa, Luigi, Yoon, John K., Dogan, Yildirim, Unnisa, Zeenath, Schindler, Jeffrey W., van Til, Niek P., Biasco, Luca, Mason, Chris
Publicado 2022

Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA

por Bugiani, Marianna, Abbink, Truus E. M., Edridge, Arthur W. D., van der Hoek, Lia, Hillen, Anne E. J., van Til, Niek P., Hu‐A‐Ng, Gino V., Breur, Marjolein, Aiach, Karen, Drevot, Philippe, Hocquemiller, Michaël, Laufer, Ralph, Wijburg, Frits A., van der Knaap, Marjo S.
Publicado 2023