Publications by authors named "C Baricordi"
Article Synopsis
- Pompe disease is a rare genetic disorder caused by a deficiency in the enzyme acid alpha-glucosidase, resulting in muscle weakness due to glycogen accumulation in lysosomes.
- Enzyme replacement therapy (ERT) is the current standard treatment but has limitations, like poor muscle penetration and immune reactions against the therapy.
- This study explores a new treatment approach using lentiviral vector-mediated gene therapy in stem cells, showing promise in reversing the disease's effects in a mouse model, along with safety assessments and insights into the treatment's mechanisms.
Background: Integration site (IS) analysis is a fundamental analytical platform for evaluating the safety and efficacy of viral vector based preclinical and clinical Gene Therapy (GT). A handful of groups have developed standardized bioinformatics pipelines to process IS sequencing data, to generate reports, and/or to perform comparative studies across different GT trials. Keeping up with the technological advances in the field of IS analysis, different computational pipelines have been published over the past decade.
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- Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) has shown promise in treating rare neurological diseases by using genetically modified microglia-like cells (MLCs) that can integrate into the brain.
- * This study explored how different administration methods impact the distribution of these MLCs and other HSPC derivatives in mice, providing important insights into their behavior and characteristics.
- * The research also found that MLCs have a distinct gene signature that differentiates them from other immune cells, and they were effective in delivering therapeutic proteins in models of Parkinson's disease and frontotemporal dementia, highlighting potential for broader treatment applications.
Our mathematical model of integration site data in clinical gene therapy supported the existence of long-term lymphoid progenitors capable of surviving independently from hematopoietic stem cells. To date, no experimental setting has been available to validate this prediction. We here report evidence of a population of lymphoid progenitors capable of independently maintaining T and NK cell production for 15 years in humans.
View Article and Find Full Text PDFAppropriate tools for monitoring sarcoma progression are still limited. The aim of the present study was to investigate the value of miR-34a-5p (miR34a) as a circulating biomarker to follow disease progression and measure the therapeutic response. Stable forced re-expression of miR34a in Ewing sarcoma (EWS) cells significantly limited tumor growth in mice.
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