Publications by authors named "Carme R Fiol"

Lentiviral vectors (LVVs) play a critical role in gene delivery for gene-modified cell therapies. However, the lack of scalable LVV production methods and the high cost associated with them may limit their use. In this work, we demonstrate the optimization and development of a scalable, chemically defined, animal component-free LVV production process using adherent human embryonic kidney 293T cells in a fixed-bed bioreactor.

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  • Ruxolitinib is the first FDA-approved treatment for myelofibrosis (MF), improving symptoms but not eliminating the disease or significantly lowering mutation levels.
  • Resistance to ruxolitinib may come from various mechanisms, allowing MF cells to activate alternative survival pathways.
  • Research identified key genes related to proteasome function that are crucial for MF cell survival, suggesting that combining proteasome inhibitors like carfilzomib with ruxolitinib could be a more effective treatment strategy for MF patients.
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Tyrosine kinase inhibitors (TKIs) targeting BCR-ABL1 have revolutionized therapy for chronic myeloid leukemia (CML), paving the way for clinical development in other diseases. Despite success, targeting leukemic stem cells and overcoming drug resistance remain challenges for curative cancer therapy. To identify drivers of kinase-independent TKI resistance in CML, we performed genome-wide expression analyses on TKI-resistant versus sensitive CML cell lines, revealing a nuclear factor-kappa B (NF-κB) expression signature.

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  • * Utilizing next-generation sequencing (NGS), researchers conducted a large-scale short hairpin RNA (shRNA) knockdown screen on nearly 5,000 genes from primary AML cells, focusing on gene knockdown across multiple timepoints for accuracy.
  • * The analysis revealed NOX1 as a critical gene with significant knockdown impact on leukaemia cell survival, making NOX family genes promising targets for developing new AML therapies.
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