Sentences with phrase «most types of gene therapy»
Unlike most types of gene therapy, a longstanding approach that aims to alter only adult human tissues that die with the patient, the Crispr technique could be used to change human eggs, sperm and early embryos, and such alterations would be inherited by the patient's children.
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«The ability to direct a gene to a specific cell type and prevent expression in other cell types is a powerful new tool that allows us to bypass one of the most troubling safety concerns facing gene therapy,» said Michael Parmacek, MD, assistant professor of medicine at the University of Chicago and director of the study.
«Our goal has been to expand the study of gene therapy beyond Type 1 infants to address the urgent medical needs of children with SMA Type 2, and we look forward to understanding the potential clinical impact of AVXS - 101 in these patients who, left untreated, will never walk on their own and most will never stand without assistance.»