Overview
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.
- | Author: Ivano Condò
- | Publisher: Mdpi Ag
- | Publication Date: Apr 27, 2023
- | Number of Pages: 250 pages
- | Language: English
- | Binding: Hardcover
- | ISBN-10: 3036574611
- | ISBN-13: 9783036574615
