Publications by authors named "F Hull"
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.
Cochlear implants and hearing aids may facilitate the development of listening and spoken language (LSL) in deaf/hard of hearing young children, but they require aural rehabilitation therapy-often unavailable outside urban areas-for optimal outcomes. This trial assessed the relative effectiveness of LSL therapy delivered either in person or by interactive video. The hypothesis was that telehealth service delivery would be noninferior to in-person therapy.
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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.