Rare Monogenic Diseases: Molecular Pathophysiology And Novel Therapies

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