Molecular Mechanisms and Determinants of Innovative Correction Approaches in Coagulation Factor Deficiencies.

ABSTRACT: Molecular strategies tailored to promote/correct the expression and/or processing of defective coagulation factors would represent innovative therapeutic approaches beyond standard substitutive therapy. Here, we focus on the molecular mechanisms and determinants underlying innovative approaches acting at DNA, mRNA and protein levels in inherited coagulation factor deficiencies, and in particular on: (i) gene editing approaches, which have permitted intervention at the DNA level through the specific recognition, cleavage, repair/correction or activation of target sequences, even in mutated gene contexts; (ii) the rescue of altered pre-mRNA processing through the engineering of key spliceosome components able to promote correct exon recognition and, in turn, the synthesis and secretion of fu

SUBMITTER: Balestra D 

PROVIDER: S-EPMC6627357 | biostudies-literature | 2019 Jun

REPOSITORIES: biostudies-literature