Research Models and Gene Augmentation Therapy for CRB1 Retinal Dystrophies

por Boon, Nanda, Wijnholds, Jan, Pellissier, Lucie P.
Publicado 2020

CRB2 Loss in Rod Photoreceptors Is Associated with Progressive Loss of Retinal Contrast Sensitivity

por Alves, C. Henrique, Boon, Nanda, Mulder, Aat A., Koster, Abraham J., Jost, Carolina R., Wijnholds, Jan
Publicado 2019

Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1(KO) and CRB1(KO)CRB2(+/−) retinal organoids

por Boon, Nanda, Lu, Xuefei, Andriessen, Charlotte A., Orlovà, Michaela, Quinn, Peter M.J., Boon, Camiel J.F., Wijnholds, Jan
Publicado 2023

Identification of long non-coding RNAs that stimulate cell survival in bladder cancer

por Dudek, Aleksandra M., Boer, Sabrina J., Boon, Nanda, Witjes, J. Alfred, Kiemeney, Lambertus A.L.M., Verhaegh, Gerald W.
Publicado 2017

AAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype

por Boon, Nanda, Lu, Xuefei, Andriessen, Charlotte A., Moustakas, Ioannis, Buck, Thilo M., Freund, Christian, Arendzen, Christiaan H., Böhringer, Stefan, Mei, Hailiang, Wijnholds, Jan
Publicado 2023

AAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype

por Boon, Nanda, Lu, Xuefei, Andriessen, Charlotte A., Moustakas, Ioannis, Buck, Thilo M., Freund, Christian, Arendzen, Christiaan H., Böhringer, Stefan, Boon, Camiel J.F., Mei, Hailiang, Wijnholds, Jan
Publicado 2023

Defining Phenotype, Tropism, and Retinal Gene Therapy Using Adeno-Associated Viral Vectors (AAVs) in New-Born Brown Norway Rats with a Spontaneous Mutation in Crb1

por Boon, Nanda, Alves, C. Henrique, Mulder, Aat A., Andriessen, Charlotte A., Buck, Thilo M., Quinn, Peter M. J., Vos, Rogier M., Koster, Abraham J., Jost, Carolina R., Wijnholds, Jan
Publicado 2021

mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy

por Riedmayr, Lisa Maria, Hinrichsmeyer, Klara Sonnie, Thalhammer, Stefan Bernhard, Mittas, David Manuel, Karguth, Nina, Otify, Dina Yehia, Böhm, Sybille, Weber, Valentin Johannes, Bartoschek, Michael David, Splith, Victoria, Brümmer, Manuela, Ferreira, Raphael, Boon, Nanda, Wögenstein, Gabriele Maria, Grimm, Christian, Wijnholds, Jan, Mehlfeld, Verena, Michalakis, Stylianos, Fenske, Stefanie, Biel, Martin, Becirovic, Elvir
Publicado 2023

CRB1 is required for recycling by RAB11A+ vesicles in human retinal organoids

por Buck, Thilo M., Quinn, Peter M.J., Pellissier, Lucie P., Mulder, Aat A., Jongejan, Aldo, Lu, Xuefei, Boon, Nanda, Koot, Daniëlle, Almushattat, Hind, Arendzen, Christiaan H., Vos, Rogier M., Bradley, Edward J., Freund, Christian, Mikkers, Harald M.M., Boon, Camiel J.F., Moerland, Perry D., Baas, Frank, Koster, Abraham J., Neefjes, Jacques, Berlin, Ilana, Jost, Carolina R., Wijnholds, Jan
Publicado 2023