4 ms·
I wonder if a similar approach can be used for Von Hippel Landau. “ Mutations in the VHL gene cause von Hippel-Lindau syndrome. The VHL gene is a tumor suppres
by mmaunder 4y ago
I wonder if a similar approach can be used for Von Hippel Landau.
“ Mutations in the VHL gene cause von Hippel-Lindau syndrome. The VHL gene is a tumor suppressor gene, which means it keeps cells from growing and dividing too rapidly or in an uncontrolled way. Mutations in this gene prevent production of the VHL protein or lead to the production of an abnormal version of the protein. An altered or missing VHL protein cannot effectively regulate cell survival and division. As a result, cells grow and divide uncontrollably to form the tumors and cysts that are characteristic of von Hippel-Lindau syndrome.”
https://medlineplus.gov/genetics/condition/von-hippel-lindau-syndrome/ https://medlineplus.gov/genetics/condition/von-hippel-lindau...
- wbl 4y agoI am not an expert but you would need much more transformation. Missing enzyme in white blood cell is simple: any unreached cells are not a problem. Defective protein in all cells is hard.
- chrisgd 4y agoI think CAR-T and cell / gene therapy is going to cure a lot of these orphan diseases with enough time. They really seem like miracles. Carl June winning a Nobel needs to be a bet on DraftKings. We have seen 10 years of remission with some ALLs in children