Key result
AAV-mediated ex-vivo genome editing triggers immune and DNA damage responses that may impair long-term cell engraftment.
Why the study?
Recent events have questioned the immunogenicity and safety profile of AAV as a gene therapy vector, but the target cells and molecular mechanisms dictating cellular responses to AAV remain poorly understood.
Design
Review
Authors
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May compromise long-term engraftment after ex-vivo HSPC editing; leaves open vector optimization to blunt immune and DNA damage responses.
Understanding the innate immune and DNA damage responses to AAV vectors in hematopoietic stem and progenitor cells is critical for improving cell survival and long-term engraftment in ex-vivo gene editing therapies.
Dudek et al. (2021) conducted a review in Ex-vivo gene editing in hematopoietic stem and progenitor cells. AAV-mediated genome editing was evaluated. AAV-mediated ex-vivo genome editing in hematopoietic stem and progenitor cells induces innate immune and DNA damage responses that may negatively impact long-term cell engraftment and clinical outcomes.
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