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Genomic Engineering in Human Hematopoietic Stem Cells: Hype or Hope?
Front Genome Ed. 2021 Jan 22;2:615619. doi: 10.3389/fgeed.2020.615619. eCollection 2020.
Front Genome Ed. 2021.
PMID: 34713237
Free PMC article.
Review.
A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies.
Zittersteijn HA, Harteveld CL, Klaver-Flores S, Lankester AC, Hoeben RC, Staal FJT, Gonçalves MAFV.
Zittersteijn HA, et al.
Front Genome Ed. 2021 Feb 4;2:617780. doi: 10.3389/fgeed.2020.617780. eCollection 2020.
Front Genome Ed. 2021.
PMID: 34713239
Free PMC article.
Review.
Item in Clipboard
Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components.
Maggio I, Zittersteijn HA, Wang Q, Liu J, Janssen JM, Ojeda IT, van der Maarel SM, Lankester AC, Hoeben RC, Gonçalves MAFV.
Maggio I, et al. Among authors: zittersteijn ha.
Gene Ther. 2020 May;27(5):209-225. doi: 10.1038/s41434-019-0119-y. Epub 2020 Jan 3.
Gene Ther. 2020.
PMID: 31900423
Free PMC article.
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A primer to gene therapy: Progress, prospects, and problems.
Zittersteijn HA, Gonçalves MAFV, Hoeben RC.
Zittersteijn HA, et al.
J Inherit Metab Dis. 2021 Jan;44(1):54-71. doi: 10.1002/jimd.12270. Epub 2020 Jul 20.
J Inherit Metab Dis. 2021.
PMID: 32510617
Free PMC article.
Review.
Item in Clipboard
Generation of dyskeratosis congenita-like hematopoietic stem cells through the stable inhibition of DKC1.
Carrascoso-Rubio C, Zittersteijn HA, Pintado-Berninches L, Fernández-Varas B, Lozano ML, Manguan-Garcia C, Sastre L, Bueren JA, Perona R, Guenechea G.
Carrascoso-Rubio C, et al. Among authors: zittersteijn ha.
Stem Cell Res Ther. 2021 Jan 29;12(1):92. doi: 10.1186/s13287-021-02145-8.
Stem Cell Res Ther. 2021.
PMID: 33514435
Free PMC article.
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