Author

R. Throm

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Sep 2026

Abstract IA06: Myogenic and neural transcriptional circuitry determines PAX3::FOXO1-driven initiation of rhabdomyosarcoma

Rhabdomyosarcoma (RMS) is the most common pediatric soft tissue sarcoma. Alveolar RMS (ARMS) has a poor prognosis and is driven by either t(2;13)(q35;q14) or t(1;13)(p36;q14) translocations resulting in the PAX3::FOXO1 (P3F) and PAX7::FOXO1 (P7F) fusion oncoproteins, respectively. Current in vivo model systems fail t...

Bradley T. Stevens, Yang E. Zhang, Matthew R. Garcia et al. · Cancer Research · 0 citations

Open access Jul 2026

Co-delivery of lentiviral vectors and Cas9-containing virus-like particles enables rapid, scalable manufacture of gene-edited CAR T cells

Chimeric antigen receptor T cell immunotherapies are transforming therapies for hematological malignancies and solid tumors and can be enhanced by targeted gene knockout. Here, we report lentiviral-based virus-like particles that package and deliver Cas9 ribonucleoproteins to primary human T cells. Using distinct pseud...

Francesca Ferrara, Matthew M Wielgosz, Jeoungeun J. Park et al. · Molecular therapy. Advances · 0 citations