Publications by authors named "R D Mazor"
Article Synopsis
- * This study focused on patients with mixed histiocytic neoplasms (MXH) and identified unique genetic mutations, while evaluating how well these patients responded to different treatments—both traditional and targeted therapies.
- * Results showed that targeted therapies significantly improved treatment outcomes, leading to higher rates of response and lower likelihood of disease progression compared to conventional therapies.
Owing to the immunogenicity of adeno-associated viruses (AAVs), gene therapies using AAVs face considerable obstacles. Here, by leveraging ex vivo T-cell assays, the prediction of epitope binding to major histocompatibility complex class-II alleles, sequence-conservation analysis in AAV phylogeny and site-directed mutagenesis, we show that the replacement of amino acid residues in a promiscuous and most immunodominant T-cell epitope in the AAV9 capsid with AAV5 sequences abrogates the immune responses of peripheral blood mononuclear cells to the chimaeric vector while preserving its functions, potency, cellular specificity, transduction efficacy and biodistribution. This rational approach to the immunosilencing of capsid epitopes promiscuously binding to T cells may be applied to other AAV vectors and epitope regions.
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- * Recent studies have identified other mutations, like those in PIK3CA, which may also drive LCH independently from MAPK signaling, although their effects were not fully understood until now.
- * New research using a mouse model shows that PIK3CA mutations can contribute to LCH, and a patient's LCH with this mutation successfully responded to a targeted treatment (alpelisib), highlighting the potential for personalized therapies in treating histiocytic neoplasms.