A primer to gene therapy: Progress, prospects, and problems

por Zittersteijn, Hidde A., Gonçalves, Manuel A.F.V., Hoeben, Rob C.
Publicado 2020

A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies

por Zittersteijn, Hidde A., Harteveld, Cornelis L., Klaver-Flores, Stefanie, Lankester, Arjan C., Hoeben, Rob C., Staal, Frank J. T., Gonçalves, Manuel A. F. V.
Publicado 2021

Genomic Engineering in Human Hematopoietic Stem Cells: Hype or Hope?

por Klaver-Flores, Stefanie, Zittersteijn, Hidde A., Canté-Barrett, Kirsten, Lankester, Arjan, Hoeben, Rob C., Gonçalves, Manuel A. F. V., Pike-Overzet, Karin, Staal, Frank J. T.
Publicado 2021

Generation of dyskeratosis congenita-like hematopoietic stem cells through the stable inhibition of DKC1

por Carrascoso-Rubio, Carlos, Zittersteijn, Hidde A., Pintado-Berninches, Laura, Fernández-Varas, Beatriz, Lozano, M. Luz, Manguan-Garcia, Cristina, Sastre, Leandro, Bueren, Juan A., Perona, Rosario, Guenechea, Guillermo
Publicado 2021

Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components

por Maggio, Ignazio, Zittersteijn, Hidde A., Wang, Qian, Liu, Jin, Janssen, Josephine M., Ojeda, Ivan Toral, van der Maarel, Silvère M., Lankester, Arjan C., Hoeben, Rob C., Gonçalves, Manuel A. F. V.
Publicado 2020