Sentences with phrase «use gene replacement»

For this we use gene replacement, live and fixed cell imaging, conditional localization and (in) activation approaches, proteomics, biochemistry and structural biology.

Not exact matches

The authors also provide experimental validation for using active genetics as an efficient means for targeted gene insertion, or «transgenesis,» and single - step replacement of genetic control elements.

Basically what was happening when you use a hematopoietic stem cell to correct an inherited metabolic disease is that through engraftment of that cell you are allowing that cell to become the replacement source for the missing enzyme or other factor - almost like a cellular form of gene therapy or, as I call it, «poor man's gene therapy».

«In our study, we used a gene therapeutic approach, but you could purify the modified protein and inject it intravenously just like it is already done for enzyme replacement therapy,» explains Spencer.

Study using disabled form of HIV in gene therapy wins highest award from Clinical Research Forum; others cited for advances revealing damage caused by long - term cortisone shots to treat arthritic knees; and gene replacement therapy for infants with deadly neuromuscular disease

Using allelic replacement, we provide functional validation of the substitution in the EBL gene controlling the growth rate in the blood stages of the parasites.

«We are really excited about this work because, short of correcting a faulty gene, protein - replacement therapy using mRNA is one of the most promising techniques we have at our disposal,» says senior author Inder Verma, professor of genetics and holder of Salk's Irwin and Joan Jacobs Chair in Exemplary Life Science.

Moreover, while still uncommonly used in the clinic, researchers have successfully developed protein replacement therapy and gene therapy for a number of coagulopathies.