Author

John P. Manis

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Open access Sep 2026

Systemic delivery of phagocytosis-shielded retroviral vectors enables in vivo HSC gene therapy for sickle cell disease

SUMMARY Ex vivo hematopoietic stem cell (HSC) gene therapy is effective for non-malignant blood disorders including sickle cell disease (SCD), but requires hospitalization, ex vivo cell manipulation, and conditioning. Direct in vivo gene delivery could remove these barriers and widen access. Here, we explore phagocytos...

Denise Klatt, Adele Mucci, Bo-Ya Liu et al. · Cell Stem Cell · 0 citations

Open access Jul 2026

Non-genotoxic transplantation and in vivo selection through epitope editing

Epitope editing of KIT enables antibody-based, non-genotoxic conditioning that selectively enriches therapeutic BCL11A-edited haematopoietic stem/progenitor cells, supports durable engraftment, preserves clonal diversity and enhances induction of fetal haemoglobin, a therapeutic approach for conditions such as sickle c...

G. Casirati, Andrea Cosentino, Marta Freschi et al. · Nature · 0 citations

#gene editing Open access Aug 2026

Analysing long-read CRISPR experiments with CRISPRLungo.

This work presents CRISPRLungo, a computational pipeline specifically designed for long-read amplicon sequencing of gene edited samples that incorporates unique molecular identifier-based error correction and statistical filtering to distinguish true editing events from background noise, enabling robust detection of sm...

Gue-Ho Hwang, Benjamin Vyshedskiy, Timothy M. Barry et al. · Nature Biomedical Engineerin... · 0 citations