Combination Therapy in Fragile X Syndrome; Possibilities and Pitfalls Illustrated by Targeting the mGluR5 and GABA Pathway Simultaneously

por Zeidler, Shimriet, de Boer, Helen, Hukema, Renate K., Willemsen, Rob
Publicado 2017

Paradoxical effect of baclofen on social behavior in the fragile X syndrome mouse model

por Zeidler, Shimriet, Pop, Andreea S., Jaafar, Israa A., de Boer, Helen, Buijsen, Ronald A. M., de Esch, Celine E. F., Nieuwenhuizen‐Bakker, Ingeborg, Hukema, Renate K., Willemsen, Rob
Publicado 2018

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

Small molecule 1a reduces FMRpolyG-mediated toxicity in in vitro and in vivo models for FMR1 premutation

por Haify, Saif N, Buijsen, Ronald A M, Verwegen, Lucas, Severijnen, Lies-Anne W F M, de Boer, Helen, Boumeester, Valerie, Monshouwer, Roos, Yang, Wang-Yong, Cameron, Michael D, Willemsen, Rob, Disney, Matthew D, Hukema, Renate K
Publicado 2021

Next Generation Sequencing Reveals Skewing of the T and B Cell Receptor Repertoires in Patients with Wiskott–Aldrich Syndrome

por O’Connell, Amy E., Volpi, Stefano, Dobbs, Kerry, Fiorini, Claudia, Tsitsikov, Erdyni, de Boer, Helen, Barlan, Isil B., Despotovic, Jenny M., Espinosa-Rosales, Francisco J., Hanson, I. Celine, Kanariou, Maria G., Martínez-Beckerat, Roxana, Mayorga-Sirera, Alvaro, Mejia-Carvajal, Carmen, Radwan, Nesrine, Weiss, Aaron R., Pai, Sung-Yun, Lee, Yu Nee, Notarangelo, Luigi D.
Publicado 2014