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Hematopoietic Stem Cell-Targeted Neonatal Gene Therapy with a Clinically Applicable Lentiviral Vector Corrects Osteopetrosis in oc/oc Mice.

November 1, 2019

Hum Gene Ther

Abstract Infantile malignant osteopetrosis (IMO) is an autosomal recessive disorder characterized by nonfunctional osteoclasts. Approximately 50% of the patients have mutations in the gene, encoding for a subunit of the osteoclast proton pump. Gene therapy represents a potential alternative treatment to allogeneic stem cell transplantation for IMO. The mouse is a model of IMO characterized […]

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Serum markers of type III and IV procollagen processing predict recurrence of fibrosis in liver transplanted patients.

October 16, 2019

Sci Rep

Abstract Following liver transplantation (LT), 10-30% of patients develop recurrent cirrhosis (RC). There is an urgent need for predictive non-invasive markers for improved monitoring of these patients. Here we studied extracellular matrix biomarkers as predictors of RC after LT. Forty-seven LT patients were divided into groups of fast, intermediate or non-progressors towards RC (5 years […]

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Serum tau fragments as predictors of death or poor neurological outcome after out-of-hospital cardiac arrest.

September 1, 2019

Biomarkers

Abstract Anoxic brain injury is the primary cause of death after resuscitation from out-of-hospital cardiac arrest (OHCA) and prognostication is challenging. The aim of this study was to evaluate the potential of two fragments of tau as serum biomarkers for neurological outcome. Single-center sub-study of 171 patients included in the Target Temperature Management (TTM) Trial […]

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Collagen type IV remodelling gender-specifically predicts mortality in decompensated cirrhosis.

May 1, 2019

Liver Int

Abstract BACKGROUND & AIMS Remodelling of extracellular matrix is crucial in progressive liver fibrosis. Collagen type III desposition has been shown in acute decompensation. Extratracellular matrix is compiled of deposition of various components. The role of basement membrane collagen type IV in advanced cirrhosis and acute decompensation is unclear and investigated in this study. METHODS […]

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