New research from the University of Pittsburgh explains why metastatic uveal melanoma is resistant to conventional immunotherapies and how adoptive therapy, which involves growing a patient's T cells outside the body before ...
Apr 16, 2024
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Findings from a recent study in the Journal of Neuromuscular Diseases demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy (SMA). The study's results add further support ...
Apr 15, 2024
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Cystic fibrosis is a genetic disease that causes serious and sometimes fatal respiratory and digestive disorders. A new treatment, available since 2020, improves lung function and quality of life. However, it does not always ...
Apr 4, 2024
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Researchers have shown that dangerous cysts, which form over time in polycystic kidney disease (PKD), can be prevented by a single normal copy of a defective gene. This means the potential exists that scientists could one ...
Apr 4, 2024
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Damage to the optic nerve can lead to irreversible blindness. A newly investigated regeneration factor could change that, UConn researchers report in the May 2024 issue of Experimental Neurology.
Mar 26, 2024
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Scientists have discovered how breast cancer cells can "hibernate" to avoid treatment and "wake up" years later—causing a relapse that is more difficult to treat.
Mar 26, 2024
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Among the many confounding symptoms in patients recovering from a COVID-19 infection are memory loss and difficulty learning. Yet little is known about the mechanisms of cognitive impairments like these, commonly called brain ...
Mar 21, 2024
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Gene therapy may be the best hope for curing retinitis pigmentosa (RP), an inherited condition that usually leads to severe vision loss and blinds 1.5 million people worldwide.
Mar 21, 2024
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Researchers at the University of Zurich and the University Hospital Zurich have discovered that a specific mutation in the cancer cells of an aggressive type of blood cancer can prevent novel immunotherapies such as CAR T-cell ...
Mar 21, 2024
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An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led ...
Mar 21, 2024
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Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, such as a hereditary disease in which a deleterious mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.
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