Results 51 to 60 of about 1,173,119 (313)
Gene Therapy for Hemophilia [PDF]
Molecular Therapy, 2016 The X-linked bleeding disorder hemophilia causes frequent and exaggerated bleeding that can be life-threatening if untreated. Conventional therapy requires frequent intravenous infusions of the missing coagulation protein (factor VIII [FVIII] for hemophilia A and factor IX [FIX] for hemophilia B).
Nienhuis, AW, Nathwani, AC, Davidoff, AM
openaire +4 more sources
FEBS Letters, EarlyView.Three‐dimensional (3D) biological systems have become key tools in lymphoma research, offering reliable in vitro and ex vivo platforms to explore pathogenesis and support precision medicine. This review highlights current 3D non‐Hodgkin lymphoma models, detailing their features, advantages, and limitations, and provides a broad perspective on future ...
Carla Faria +3 more
wiley +1 more source
Early induction of cytokine release syndrome by rapidly generated CAR T cells in preclinical models
EMBO Molecular MedicineCytokine release syndrome (CRS) is a significant side-effect of conventional chimeric antigen receptor (CAR) T-cell therapy. To facilitate patient accessibility, short-term (st) CAR T cells, which are administered to patients only 24 h after vector ...
Arezoo Jamali +5 more
doaj +1 more source
Gene therapy for arthritis [PDF]
Modern Rheumatology, 2008 Arthritis is among the leading causes of disability in the developed world. There remains no cure for this disease and the current treatments are only modestly effective at slowing the disease's progression and providing symptomatic relief. The clinical effectiveness of current treatment regimens has been limited by short half-lives of the drugs and ...
Raphael Hirsch, Russell S. Traister
openaire +3 more sources
From omics to AI—mapping the pathogenic pathways in type 2 diabetes
FEBS Letters, EarlyView.Integrating multi‐omics data with AI‐based modelling (unsupervised and supervised machine learning) identify optimal patient clusters, informing AI‐driven accurate risk stratification. Digital twins simulate individual trajectories in real time, guiding precision medicine by matching patients to targeted therapies.
Siobhán O'Sullivan +2 more
wiley +1 more source
BMC Genomics, 2020 An amendment to this paper has been published and can be accessed via the original article.
Camilo Breton +4 more
doaj +1 more source
FEBS Letters, EarlyView.C‐mannosylation is a unique form of protein glycosylation. In this study, we demonstrated that ADAMTS1 is C‐mannosylated at Trp562 and Trp565 in human testicular germ cell tumor NEC8 cells. We found that C‐mannosylation of ADAMTS1 is essential for its secretion, processing, enzymatic activity, and ability to promote vasculogenic mimicry. These findings
Takato Kobayashi +5 more
wiley +1 more source
Gene Therapy for Hemoglobinopathies [PDF]
Human Gene Therapy, 2018 Gene therapy for β-thalassemia and sickle-cell disease is based on transplantation of genetically corrected, autologous hematopoietic stem cells. Preclinical and clinical studies have shown the safety and efficacy of this therapeutic approach, currently based on lentiviral vectors to transfer a β-globin gene under the transcriptional control of ...
Cavazzana M., Mavilio F.
openaire +3 more sources
FEBS Letters, EarlyView.Spinal muscular atrophy (SMA) is a genetic disease affecting motor neurons. Individuals with SMA experience mitochondrial dysfunction and oxidative stress. The aim of the study was to investigate the effect of an antioxidant and neuroprotective substance, ergothioneine (ERGO), on an SMNΔ7 mouse model of SMA.
Francesca Cadile +8 more
wiley +1 more source
Stem Cell Research & Therapy, 2016 Background Cirrhosis is a major health problem worldwide and new therapies are needed. Hepatic macrophages (hMø) have a pivotal role in liver fibrosis, being able to act in both its promotion and its resolution.
Esteban Fiore +10 more
doaj +1 more source