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Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency.
Commun Med (Lond)
January 2025
Rare Disease Translational Center, The Jackson Laboratory, Bar Harbor, ME, USA.
Background: Multiple Sulfatase Deficiency (MSD) is a rare inherited lysosomal storage disorder characterized by loss of function mutations in the SUMF1 gene that manifests as a severe pediatric neurological disease. There are no available targeted therapies for MSD.
Methods: We engineered a viral vector (AAV9/SUMF1) to deliver working copies of the SUMF1 gene and tested the vector in Sumf1 knock out mice that generally display a median lifespan of 10 days.
Dual-task-related gait patterns as possible marker of precocious and subclinical cognitive alterations in Parkinson disease.
Sci Rep
January 2025
Department of Medicine, Surgery and Dentistry, Center for Neurodegenerative Diseases (CEMAND), University of Salerno, Fisciano, Italy.
Subtle gait and cognitive dysfunction are common in Parkinson's disease (PD), even before most evident clinical manifestations. Such alterations can be assumed as hypothetical phenotypical and prognostic/progression markers. To compare spatiotemporal gait parameters in PD patients with three cognitive status: cognitively intact (PD-noCI), with subjective cognitive impairment (PD-SCI) and with mild cognitive impairment (PD-MCI) in order to detect subclinical gait differences.
View Article and Find Full Text PDFHexahistidine-metal assembly encapsulated fibroblast growth factor 21 for lipopolysaccharide-induced acute lung injury.
Eur J Pharm Biopharm
January 2025
Intervention Department, The First Affiliated Hospital of Wenzhou Medical University, Wenzhou, Zhejiang 325000, China. Electronic address:
Acute lung injury (ALI)/acute respiratory distress syndrome (ARDS) represents a spectrum of potentially fatal conditions that currently lack effective drug treatment. Recent researches suggest that Fibroblast Growth Factor 21 (FGF21) may protect against ALI/ARDS. However, the clinical use of FGF21 is limited by its rapid degradation, restricted targeting capabilities, and numerous adverse effects.
View Article and Find Full Text PDFThe oral and maxillofacial manifestations of Stickler syndrome: a systematic review.
J Stomatol Oral Maxillofac Surg
January 2025
Univ. Lille, CHU Lille, Oral and Maxillofacial Surgery Department, Lille, France.
Introduction: Stickler syndrome is a rare genetic collagen disorder known for its ophthalmological abnormalities. However, there are several other associated facial features. The aim of this study is to review the literature on the various oral and maxillofacial manifestations of Stickler syndrome.
View Article and Find Full Text PDFAssessment and management for latent tuberculosis before advanced therapies for immune-mediated inflammatory diseases: A comprehensive review.
Autoimmun Rev
January 2025
Department of Gastroenterology, Postgraduate Institute of Medical Education and Research, Chandigarh, India. Electronic address:
Tuberculosis (TB), caused by Mycobacterium TB, is the most significant infectious cause of mortality across the globe. While TB disease can prey on immunocompetent individuals, it is more likely to occur in immunocompromised individuals. Immune-mediated inflammatory diseases (IMIDs) are a group of diseases (rheumatoid arthritis, inflammatory bowel disease, ankylosing spondylitis, psoriasis, hidradenitis suppurative, autoimmune blistering diseases, and others) where there may be a need for systemic immunosuppression to control the disease manifestations, treat symptoms and improve long term outcomes.
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