Rare Monogenic Diseases

<p>The reprint Rare Monogenic Diseases: Molecular Pathophysiology and Novel Therapies provides a collection of original research articles and systematic reviews focused on diverse conditions stemming from a pathogenic single gene mutation. The articles are focused on the molecular mechanisms that are affected within a rare monogenic disorder such as those involving gene expression molecular pathways and cell death. Moreover specific experimental therapies including drug discovery drug repositioning gene therapy and protein-based therapeutics are discussed.</p>