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Gene therapy can effectively treat mice with tuberous sclerosis complex, shows study – News-Medical.Net
Patients with tuberous sclerosis complex, a genetic disorder characterized by the growth of noncancerous tumors in multiple organs of the body, have limited treatment…
Patients with tuberous sclerosis complex, a genetic disorder characterized by the growth of noncancerous tumors in multiple organs of the body, have limited treatment options. A team led by investigators at Massachusetts General Hospital (MGH) has now shown that gene therapy can effectively treat mice that express one of the mutated genes that cause the disease. The research is published in Science Advances.
The gene, called TSC2, codes for tuberin, a protein that acts to inhibit cell growth and…
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