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Rasmus O. Bak

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#gene editing Sep 2026

Prime editing of human hematopoietic stem cells for correction of GATA2 deficiency.

GATA2 (GATA binding protein 2) deficiency is a severe immunodeficiency caused by heterozygous variants in the gene encoding the transcription factor GATA2. Ex vivo gene editing of a patient's own CD34+ hematopoietic stem and progenitor cells (HSPCs) could provide curative treatment. However, current methods that rely o...

J. H. Wolff, T. W. Skov, S. R. Dorset et al. · 0 citations
#gene editing Open access Sep 2026

Clinical Applications of In Vivo Gene Editing: Methods, Challenges, and Solutions

In this course, we investigate into the complex landscape of gene editing within aclinical framework, focusing on the generation of a comprehensive map detailingcurrent delivery methodologies employed for in vivo gene editing across variousanimal models and clinical scenarios, with some insight for ex vivo studies. The...

Yonglun Luo, Karim Benabdellah, Mariana Köber et al. · 0 citations
#gene editing Open access Sep 2026

Clinical Applications of In Vivo Gene Editing: Methods, Challenges, and Solutions

In this course, we investigate into the complex landscape of gene editing within aclinical framework, focusing on the generation of a comprehensive map detailingcurrent delivery methodologies employed for in vivo gene editing across variousanimal models and clinical scenarios, with some insight for ex vivo studies. The...

Yonglun Luo, Karim Benabdellah, Mariana Köber et al. · 0 citations

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