A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy

A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy

Mutations within the fukutin-related protein (FKRP) gene end in a broad spectrum of muscular dystrophy (MD) phenotypes, together with the extreme Walker-Warburg syndrome (WWS). Right here, we develop a gene-editing method that replaces your complete mutant open studying body with the wild-type sequence to universally right all FKRP mutations. We apply this method to right […]

Preclinical safety evaluation of a recombinant plasmid vector encoding mature human neutrophil peptide-1 by repeated local administrations in nonhuman primates

Preclinical safety evaluation of a recombinant plasmid vector encoding mature human neutrophil peptide-1 by repeated local administrations in nonhuman primates

In our earlier research, a novel gene remedy method was developed based mostly on a plasmid vector pSecTag2B through which recombinant HNP1 gene was regulated below a cytomegalovirus promoter to encode a mature HNP1 type. We confirmed for the primary time in numerous tumor fashions together with human most cancers xenografts that overexpression of HNP1 […]