ArticleMolecular therapy. Methods & clinical development2025
Bedside manufacturing of engineered stem cells using gene therapy foam.
Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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4 authors.
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Abstract
Hematopoietic stem cell (HSC) gene therapies hold immense potential for treating a growing list of genetic disorders, but the field has reached an inflection point where novel technologies are needed to overcome significant challenges, including high costs, unequal access, and prolonged disruption of daily life. Here, we describe a bedside cell manufacturing strategy in which HSCs are genetically targeted within gene therapy foam that is mixed with freshly isolated bone marrow aspirate concentrate and then directly injected back into the marrow. We demonstrate that highly efficient genetic reprogramming of CD34+ HSCs can be accomplished within methylcellulose-based foam, using low vector doses that are inefficient with conventional liquid-based approaches. In an
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