Sentences with phrase «through gene replacement»

Gene therapy is a promising approach for treating a large spectrum of human diseases and particularly monogenic diseases that can be corrected through gene replacement therapy based on introducing a functional copy of the defective gene.

Not exact matches

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».

This genome was subsequently converted into a non-functional form through replacement of the essential «rrs» gene (that encodes for the 16S rRNA) with a ura3 yeast marker.