91社区

My training was in Genetics at University College, London, UK. Protein trafficking diseases are the current focus of our group. In particular, the mechanism of correction of the cellular trafficking defects of mutant proteins. Cystic Fibrosis (CF) is a protein trafficking disease where the mutant, but otherwise functional proteins recognized by the cellular quality control system. We have identified genes that correct the trafficking defect of mutant CFTR. We have shown that the inactive versions of these genes are the active agents in correcting the trafficking of mutant CFTR. The novelty of this approach is that it is the inactive versions of the two genes that correct the trafficking of mutant CFTR. We have shown that the shRNA in lentivirus versions of these genes can also efficiently correct mutant CFTR trafficking. We are currently refining in cells from cystic fibrosis patients, their potential for a new approach for CF gene therapy.

听