Author

Anna K. Anderson

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Review Open access Sep 2026

Host Barriers to In Vivo HSC Gene Therapy with Helper-Dependent Adenovirus Vectors

Ex vivo hematopoietic stem cell (HSC) gene therapy strategies have proved effective for the treatment of various diseases, with recent advances leading to the clinical approval of several cell products. To overcome issues associated with the cost and technical complexity of ex vivo strategies, we turn towards in vivo s...

Anna K. Anderson, André Lieber, Chang Li · Viruses · 0 citations

#gene editing Open access Sep 2026

In vivo HSPC gene therapy of hemoglobinopathies without drug selection of corrected cells

In vitro studies in an erythroid progenitor cell line and primary CD34+ cells demonstrated that the EPORW439* variant conferred a strong proliferative advantage to therapeutically modified erythroid progenitors, establishing a strategy to amplify the therapeutic benefit of otherwise limited in vivo HSPC gene editing fo...

Akshara Sakunthala Velmurugan, K. Paschoudi, Jack A. Queenan et al. · bioRxiv · 0 citations