Therapeutic administration of interleukin-10 (IL-10) into the cerebrospinal fluid through intrathecal gene therapy caused fatal meningitis in swine, an unexpected toxicity that had not been seen in multiple rodent experiments.
As far as gene editing technologies go, clustered regularly interspaced short palindromic repeats, or CRISPR, tends to suck up all the oxygen in the room.
WASHINGTON – "There is nothing more beautiful than 20/20 hindsight," Huda Zoghbi told the audience at the 2017 Annual Meeting of the Society for Neuroscience.
WASHINGTON – "In the 21st century, there is not a single cure for a single neurodegenerative disease. Not one. . . . There are no therapeutics. There is no strategy. There is bupkes in the 21st century."
Fibroblasts are the major cell type that is responsible for wound healing, as they migrate to the sites of injuries and secrete extracellular matrix molecules that physically allow repair of damaged tissue.
Transgenic skin stem cells combined with improved tissue engineering methods have enabled an almost total autologous skin transplant for a 7-year-old child with junctional epidermolysis bullosa (JEB), a rare genetic disease that causes the top layer of skin, the epidermis, to separate from the underlying dermis.
Treating aging rats with an experimental compound that prevented neuronal cell death protected the animals from both depressive symptoms that are often the first symptom of Alzheimer’s disease (AD), and the memory decline that follows. The compound did not, however, affect the amyloid beta (a-beta) plaques, tau phosphorylation and inflammation that are widely considered to be the toxic agents behind neuronal demise in AD.