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Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency.
Mol Ther
November 2024
Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Human Genetics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Electronic address:
Article Synopsis
- Multiple sulfatase deficiency (MSD) is a serious disorder where the body's cells can't break down certain substances due to a problem with a gene called SUMF1.
- Researchers tested a new treatment using gene therapy and stem cell transplants in mice with MSD to see if it could help improve their condition.
- The treatment showed positive results by boosting important protein levels and improving brain function and health in the affected mice.
Paralytic Shellfish Toxins in Mollusks from Galicia Analyzed by a Fast Refined AOAC 2005.06 Method: Toxicity, Toxin Profile, and Inter-Specific, Spatial, and Seasonal Variations.
Toxins (Basel)
May 2024
Instituto Tecnolóxico para o Control do Medio Mariño de Galicia (Intecmar), Vilagarcía de Arousa, 36611 Pontevedra, Spain.
Paralytic shellfish poisoning is an important concern for mollusk fisheries, aquaculture, and public health. In Galicia, NW Iberian Peninsula, such toxicity has been monitored for a long time using mouse bioassay. Therefore, little information exists about the precise toxin analogues and their possible transformations in diverse mollusk species and environments.
View Article and Find Full Text PDFThe Utility and Safety of a Continuous Glucose Monitoring System (CGMS) in Asphyxiated Neonates during Therapeutic Hypothermia.
Diagnostics (Basel)
September 2023
Department of Woman and Child Health and Public Health, Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.
Background: The present study was designed to assess the feasibility and reliability of a Continuous Glucose Monitoring System (CGMS) in a population of asphyxiated neonates during therapeutic hypothermia.
Methods: This non-randomized feasibility study was conducted in the Neonatal Intensive Care Unit (NICU) facilities of Fondazione Policlinico A. Gemelli IRCSS.
In vivo hematopoietic stem cell modification by mRNA delivery.
Science
July 2023
Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Hematopoietic stem cells (HSCs) are the source of all blood cells over an individual's lifetime. Diseased HSCs can be replaced with gene-engineered or healthy HSCs through HSC transplantation (HSCT). However, current protocols carry major side effects and have limited access.
View Article and Find Full Text PDFTop-Down System for Multi-Person 3D Absolute Pose Estimation from Monocular Videos.
Sensors (Basel)
May 2022
Alqualsadi Research Team, Rabat IT Center, ENSIAS, Mohammed V University in Rabat, Rabat 10112, Morocco.
Two-dimensional (2D) multi-person pose estimation and three-dimensional (3D) root-relative pose estimation from a monocular RGB camera have made significant progress recently. Yet, real-world applications require depth estimations and the ability to determine the distances between people in a scene. Therefore, it is necessary to recover the 3D absolute poses of several people.
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