A virus-like particle (VLP)-based toolkit that delivers diverse CRISPR editing modalities to human monocytes, macrophages and dendritic cells with high efficiency while preserving viability and innate immune responsiveness is presented.
Hyuncheol Jung, Pascal Devant, Carter Ching et al.· Nature Biotechnology· 0 citations
A proof-of-concept study supports the promise of CD4-NBs as a minimally invasive, CD4⁺ cell-targeted gene editing strategy for HIV therapy and reduces viremia in ART-pretreated humanized mice.
Jolien Van Cleemput, Maaike De Cock, R. Verbeek et al.· EMBO Molecular Medicine· 0 citations
Lipofection- and lentivirus-mediated protocols for CRISPR-Cas9 delivery in mouse-passaged primary human hepatocytes (mpPHH) are reported, a system that enables PHH expansion in liver-humanized mice and enables scalable genetic manipulation of mpPHH, opening new avenues for HBV research and liver disease modeling.
Ansgar F. Stenzel, Antonis Athanasiadis, Georgios Dangas et al.· bioRxiv· 0 citations
Human induced pluripotent stem cells (hiPSCs) represent a powerful platform for disease modeling, especially in monogenic diseases as they preserve the donor’s genetic background while enabling directed differentiation into disease-relevant cell types. This makes them highly suitable for studying disease mechanisms in...
D. Skoczek, J. Hohendorff, Maciej T. Małecki et al.· Human Genetics· 0 citations
REPLACE is established as a versatile platform for homology-independent sequence replacement and as a complementary approach for genetic correction in settings where homology-directed repair (HDR), base editing (BE) or prime editing (PE) are inefficient or not applicable.
Tu Dang, Alexandra Roman, Anja Zimmer et al.· bioRxiv· 0 citations
It is concluded from preliminary data that gene therapy using BE HSPCs appear very promising for achieving an earlier and more robust multi-lineage immune reconstitution in adult patients using BE HSPCs.
S. S. de Ravin, Michelle Ma, Yuzhi Yin et al.· Journal of Human Immunity· 0 citations
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