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Inflammation plays a considerable role in the progression of Duchenne muscular dystrophy ( DMD ), a severe muscle disease caused by a mutation in the dystrophin gene. Researchers previously showed ...


Deregulation and inhibition of the immune system contributes to cancer development. Many therapeutic strategies aim to re-stimulate the immune system to recognize cancer cells and target them for dest ...


The functional aspects of programmed death 1 ( PD-1 ) and PD ligand 1 ( PD-L1 ) immune checkpoints in malignant mesothelioma have not been studied. Tumor samples from 65 patients with mesothelioma ...


Metachromatic leukodystrophy ( MLD ) is a lysosomal storage disease caused by a functional deficiency of the lysosomal enzyme arylsulfatase A. The prevailing late-infantile variant of metachromatic ...


Findings from an ongoing phase IIa study to evaluate the safety and efficacy of investigational chimeric antigen receptor T cell ( CART ) therapy CTL019 in certain types of relapsed or refractory ( r/ ...


The latest findings from an ongoing phase II study of CTL019, an investigational chimeric antigen receptor T cell ( CART ) therapy, further support its potential in the treatment of children and young ...


Comparative studies suggest that stem cells committed to a cardiac lineage are more effective for improving heart function than those featuring an extra-cardiac phenotype. Researchers have therefore ...


Neurodegenerative diseases such as Alzheimer’s are associated with the aggregation of endogenous peptides and proteins that contribute to neuronal dysfunction and loss. The glymphatic system, a brai ...


Gene therapy for Leber congenital amaurosis ( LCA ), an inherited disorder that causes vision loss starting in childhood, improved patients’ eyesight and the sensitivity of the retina within weeks of ...


Using brain tumor samples collected from children in the United States and Europe, a team of researchers found that the drug Panobinostat ( Farydak ) and similar gene regulating drugs may be effective ...


PARP-1 and PARP-2 are nuclear enzymes activated by DNA strand breaks and are involved in recruitment of DNA repair proteins to sites of damage. PARP-1 and PARP-2 share partially overlapping functions, ...


Lung cancer is the number one cause of cancer mortality globally and has an estimated incidence of 1.3 million new cases every year. Approximately 80–85% of the newly diagnosed cases of lung cancer ar ...


Immune checkpoint inhibitors are effective cancer treatments, but molecular determinants of clinical benefit are unknown. Ipilimumab ( Yervoy ) and Tremelimumab are antibodies against cytotoxic T-lymp ...


Therapies that target the programmed death-1 ( PD-1 ) receptor have shown unprecedented rates of durable clinical responses in patients with various cancer types. One mechanism by which cancer tissues ...


Patients with multiple sclerosis were able to safely tolerate treatment with cells cultured from human placental tissue, according to a study published in the journal Multiple Sclerosis and Related Di ...