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Preclinical evaluation of the efficacy and safety of AAV8-TNAP-D10 in Alpl-/- and AlplPrx1/Prx1 mouse models for the treatment of early and late-onset hypophosphatasia.
J Bone Miner Res
January 2025
Human Genetics Program, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA, United States.
We previously documented successful resolution of skeletal and dental disease in the infantile and late-onset murine models of hypophosphatasia (HPP), with a single injection of an adeno-associated serotype 8 vector encoding mineral-targeted TNAP (AAV8-TNAP-D10). Here, we conducted dosing studies in both HPP mouse models. A single escalating dose from 4x108 up to 4x1010 (vg/b) was intramuscularly injected into 4-day-old Alpl-/- mice (an infantile HPP model) and a single dose from 4x106 up to 4x109 (vg/b) was administered to 8-week-old AlplPrx1/Prx1 mice (a late-onset HPP model).
View Article and Find Full Text PDFTechnical Advances Drive the Molecular Understanding of Effectors from Wheat and Barley Powdery Mildew Fungi.
Mol Plant Microbe Interact
January 2025
University of Cologne, Institute for Plant Sciences, Cologne, Germany.
Pathogens manipulate host physiology through the secretion of virulence factors (effectors) to invade and proliferate on the host. The molecular functions of effectors inside plant hosts have been of interest in the field of molecular plant-microbe interactions. Obligate biotrophic pathogens, such as rusts and powdery mildews, cannot proliferate outside of plant hosts.
View Article and Find Full Text PDFYoung Adults with Type 1 Diabetes' Clinical Outcomes and Satisfaction Related to the Use of Videoconferencing for Type 1 Diabetes Healthcare: A Narrative Review.
Diabetes Ther
January 2025
Departamento de Endocrinología y Metabolismo, Unidad de Investigación en Enfermedades Metabolicas, Instituto Nacional de Ciencias Médicas y Nutrición, Salvador Zubirán, Mexico City, Mexico.
Introduction: Young adulthood is well documented as being a particularly challenging area of type 1 diabetes (T1D) healthcare. Many young adults with T1D (YAT1D) are distracted from effective disease self-management; T1D healthcare service engagement can be problematic and inconsistent, and high rates of unplanned healthcare contacts prevail. Video conferencing use can facilitate services to be flexible and responsive.
View Article and Find Full Text PDFA Review of Falls And Injuries Of Nursing Home Residents Presenting To The Emergency Department.
QJM
January 2025
Tallaght hospital, Dept. of Age Related Healthcare; Trinity College Dublin, Dept. of Medical Gerontology.
Background: Falls are frequently reported within the HSE. The Irish Longitudinal Study on Ageing(TILDA) found that 40% of over 50 s experience a fall in a two year period, with 20% requiring hospital attendance (1). It has been estimated that the cost of injuries related to falls in older people will increase exponentially over the coming years (2).
View Article and Find Full Text PDFMaking the Case for an International Childhood Cancer Data Partnership.
J Natl Cancer Inst
January 2025
Surveillance Research Program, Division of Cancer Control and Population Sciences, National Cancer Institute, Rockville, MD, USA.
Childhood cancers are a heterogeneous group of rare diseases, accounting for less than 2% of all cancers diagnosed worldwide. Most countries, therefore, do not have enough cases to provide robust information on epidemiology, treatment, and late effects, especially for rarer types of cancer. Thus, only through a concerted effort to share data internationally will we be able to answer research questions that could not otherwise be answered.
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