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Patients with tuberous sclerosis complex, a genetic disorder characterized by the growth of noncancerous tumors in several organs of the body, have limited treatment choices. A team led by investigators at Massachusetts General Hospital (MGH) has shown that gene therapy can effectively treat mice that express one of the mutated genes that cause the disease. The study is published in Science Advances.
The gene, known as TSC2, codes for tuberin, a protein that acts to inhibit cell growth and proliferation. When mutations occur in TSC2, resulting in a lack of tuberin in cells, the cells expand and multiply, leading to the formation of tumors.
Mice with tuberous sclerosis complex had a shortened life span of about 58 days on average, and they showed signs of brain abnormalities consistent with those which are often seen in patients with the illness. When the mice were injected intravenously with the gene therapy treatment, however, their average survival was extended to 462 days, and their brains revealed decreased signs of damage.
Current treatments for tuberous sclerosis complex include surgery and/or lifelong treatment with drugs that cause immune suppression and potentially compromise early brain development. Therefore, there is a clear need to identify other therapeutic approaches for this disease. Adeno-associated virus vectors have been used widely in clinical trials for many hereditary diseases with little to no toxicity, long-term action in nondividing cells, and improvement in symptoms.”
Shilpa Prabhakar, Co-Lead Author, Investigator, MGH Departments of Neurology and Radiology
She notes that benefits can be seen following a single injection, and some forms of the viral vector can efficiently enter the brain and peripheral organs following intravenous injection.
The U.S. Food and Drug Administration has approved a limited number of gene therapy products for use in humans, and the results from this research suggest that clinical trials are warranted to check the strategy’s potential in patients with tuberous sclerosis complex.
Massachusetts General Hospital
Cheah, P-S., et al. (2021) Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin. Science Advances. doi.org/10.1126/sciadv.abb1703.