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Is there a gene therapy cure for neurofibromatosis?

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The study, from the Washington University School of Medicine in St. Louis, MO, is published in the journal Human Molecular Genetics. read more

There is currently no cure for Neurofibromatosis type1 And there are no medications to help treat it. This page may be out of date. Save your draft before refreshing this page. read more

One possible preventive treatment for NF1 would involve gene therapy. We hypothesize that it is possible that a plasmid vector for this gene therapy would be preferable to a viral vector, because it would avoid the problems of inactivation of other genes by random insertion, and activation of the immune system. read more

Gene therapy is an unlikely solution as treatment due to the large size of the NF gene. I'm doubtful there is a vactor large enough to carry the entire NF gene and deliver to the correct site in all cells in the body. No one as far as I know is studying or researching into gene therapy meant to replace the entire NF gene. read more

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