Author

Donald B. Kohn

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Open access Sep 2026

APOBEC3 Knockout in Lentiviral Packaging Cells Improves Therapeutic Transgene Sequence Fidelity.

Lentiviral vectors are a well-established method of delivering transgenes into target cells. Lentiviral vectors have a strong efficacy and safety profile, but transgene fidelity remains an area of concern. Loss of function mutations within the integrated transgene may reduce therapeutic efficacy. Gain of function mutat...

Annika Mittelhauser, R. Patel, Alex B. Costa et al. · Molecular Therapy · 0 citations

#gene editing Open access Sep 2026

Development of CRISPR_SCD001, an Autologous Hematopoietic Stem Cell Gene Therapy for Sickle Cell Disease after CRISPR-Cas9 Mediated Correction.

Data support initiation of a first-in-human phase I trial of CRISPR_SCD001 for sickle patients presenting with severe pathology, and generates a clinical-grade, cryopreserved CD34+ cell product that corrects the sickle mutation, restores anti-sickling hemoglobins, and meets pre-clinical safety criteria.

M. DeWitt, Beatriz Campo-Fernández, Sohini Roy et al. · Molecular Therapy · 0 citations

Open access Aug 2026

Double-stranded DNA donors and CRISPR-Cas9 for universal correction of mutations causing cystic fibrosis in human airway cells

These results demonstrate that low levels of CFTR integration can be made therapeutically relevant by optimizing the designs of gene editing reagents, an essential step toward in vivo gene therapy for CF.

Vrishti Sinha, P. Ayoub, C. Juett et al. · Molecular Therapy: Nucleic A... · 0 citations