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

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

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 acti...

Full description

Bibliographic Details
Main Authors: Dario Balestra, Alessio Branchini
Format: Article
Language:English
Published: MDPI AG 2019-06-01
Series:International Journal of Molecular Sciences
Subjects:
coagulation factor deficiencies
gene therapy
TALEs
CRISPR activation
RNA-based correction approaches
modified U1 snRNA
ribosome readthrough
chaperone-like compounds
Online Access:https://www.mdpi.com/1422-0067/20/12/3036

Internet

https://www.mdpi.com/1422-0067/20/12/3036

Similar Items