Publications by authors named "Lindsey Skrdlant"

Article Synopsis
  • T cell receptors (TCRs) are crucial for T cells to detect cancer cell mutations, and researchers used a CRISPR-Cas9 method to edit TCR genes in a clinical trial setting.
  • Sixteen patients with advanced solid cancers received personalized T cell therapies featuring engineered neoTCRs, with most participants experiencing either stable disease or disease progression.
  • The study confirmed that it is feasible to create multiple engineered TCRs, showing the safety and effectiveness of infusing gene-edited T cells that can successfully target tumors.
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Lentiviral vectors are a common tool used to introduce new and corrected genes into cell therapy products for treatment of human diseases. Although lentiviral vectors are ideal for delivery and stable integration of genes of interest into the host cell genome, they potentially pose risks to human health, such as integration-mediated transformation and generation of a replication competent lentivirus (RCL) capable of infecting non-target cells. In consideration of the latter risk, all cell-based products modified by lentiviral vectors and intended for patient use must be tested for RCL prior to treatment of the patient.

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Background: Serine-arginine rich splicing factor 2 (SRSF2) is a protein known for its role in RNA splicing and genome stability. It has been recently discovered that SRSF2, along with other splicing regulators, is frequently mutated in patients with myelodysplastic syndrome (MDS). The most common MDS mutations in SRSF2 occur at proline 95; the mutant proteins are shown to have different RNA binding preferences, which may contribute to splicing changes detected in mutant cells.

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SR proteins are a class of RNA-binding proteins whose RNA-binding ability is required for both constitutive and alternative splicing. While members of the SR protein family were once thought to have redundant functions, in-depth biochemical analysis of their RNA-binding abilities has revealed distinct binding profiles for each SR protein, that often lead to either synergistic or antagonistic functions. SR protein family members SRSF1 and SRSF2 are two of the most highly studied RNA-binding proteins.

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