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A multiple targeting rapamycin and SS31 conjugate enhances ischemic stroke therapy.
Expert Opin Drug Deliv
January 2025
Wuya College of Innovation, Shenyang Pharmaceutical University, Shenyang, China.
Background: The identification of drugs targeting multiple pathways is essential for comprehensive protection against cerebral ischemia-reperfusion injury.
Research Design And Methods: This study aimed to develop RS31, a multi-target cytoprotectant composed of SS31 (an oxidative stress mitigator) and rapamycin (Rapa), contributes anti-inflammatory and blood-brain barrier protection. RS31 was synthesized using click chemistry, and its ability to scavenge reactive oxygen species (ROS) and reduce inflammation was tested in HO-injured PC12 cells and LPS-stimulated BV2 cells.
Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease.
N Engl J Med
November 2024
From the University of California, San Diego Medical Center, La Jolla (B.G., E.A.); University of Colorado Anschutz Medical Campus, Aurora (M.T.); Boston's Children's Hospital, Harvard Medical School, Boston (S.C.); Rocket Pharmaceuticals, Cranbury, NJ (D.R., P.Y., P.B., G.S., K.P., M.C., S.C.-K., J.D.S.); and Children's Hospital of Philadelphia and Perelman School of Medicine, University of Pennsylvania (J.W.R.) - both in Philadelphia.
Background: Danon disease is a rare, X-linked, monogenic cardiomyopathy caused by mutations in the lysosomal-associated membrane 2 gene (), which encodes the LAMP2 protein. In male patients, the predominant phenotype is progressive cardiac hypertrophy, cardiac dysfunction, and early death. There are no directed therapies for the disease.
View Article and Find Full Text PDFIn-human clinical experience with direct stick embolization of low-flow vascular malformations using a mammalian target of rapamycin inhibitor.
J Vasc Surg Venous Lymphat Disord
November 2024
The Vascular Care Group (TVCG), Darien, CT; Vascular Anomalies and Malformations Program (VAMP), Yale School of Medicine, New Haven, CT; Division of Vascular Surgery, Department of Surgery, Yale New Haven Health, New Haven, CT. Electronic address:
Background: Although direct stick embolization (DSE) of low-flow vascular malformations (LFVMs) with off-label embolotherapeutic compounds is the current mainstay of therapy, systemic oral mammalian target of rapamycin (mTOR) inhibition has evolved into an important adjunctive therapy that is associated with frequent blood draws, systemic toxicity, and rebound signs and symptoms upon cessation. We herein report our experience with in-human DSE of LFVMs with an mTOR inhibitor for direct, intralesional targeting of the culprit mutated pathway without repeated systemic exposure.
Methods: Since 2020, 33 procedures involving DSE were performed in 25 patients with LFVMs using a patented formulation and technique involving the intravenously compatible mTOR inhibitor Yale-OCR7737, used as a liquid compound in a collagen matrix emulsion for added viscosity and intralesional residence.
Thioredoxin-1 protein interactions in neuronal survival and neurodegeneration.
Biochim Biophys Acta Mol Basis Dis
January 2025
Department of Physiology and Pathophysiology, University of Manitoba, Canada. Electronic address:
Neuronal cell death remains the principal pathophysiologic hallmark of neurodegenerative diseases and the main challenge for treatment strategies. Thioredoxin1 (Trx1) is a major cytoplasmic thiol oxidoreductase protein involved in redox signaling, hence a crucial player in maintaining neuronal health. Trx1 levels are notably reduced in neurodegenerative diseases including Alzheimer's and Parkinson's diseases, however, the impact of this decrease on neuronal physiology remains largely unexplored.
View Article and Find Full Text PDFSturge-Weber syndrome: an overview of history, genetics, clinical manifestations, and management.
Semin Pediatr Neurol
October 2024
The University of Texas at Austin, Dell Medical School, Department of Neurology, 1601 Trinity St., Bldg. B, Stop Z0700 Austin, TX 78712, USA; Dell Children's Medical Center, 4910 Mueller Blvd Suite 300 Austin, TX 78723, USA. Electronic address:
Article Synopsis
- * Neurological issues associated with the syndrome include epilepsy, stroke-like episodes, and cognitive impairment, with MRI typically showing characteristic brain changes.
- * Treatment options involve surgery for glaucoma and laser therapy for port-wine stains, along with potential benefits from anti-seizure medications and low-dose aspirin for managing symptoms.
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