Elimination of the B4Galnt1 gene normalizes lifespan and prevents pathology in Tay-Sachs disease micedoi:10.1016/j.ymgme.2022.107315Volkan SeyrantepeElsevier Inc.Molecular Genetics and Metabolism
Direct injection to the brain of several viral vectors encoding either HEXA or HEXB or both seemed to yield impressive improvements in brain GM2 and extended the lifespan of mice with the rapidly progressing and lethal Sandhoff’s disease if injected early enough in the post-natal weeks (26–...
But when TNFa was subsequently removed, there was a significant improvement in the lifespan of themiceandneurological function. "We also found that neurons didn't die as early as they do with the disease, so we delayed the progression as well," says Igdoura. "We have identified a molecule...