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Mdpi AG

Rare Monogenic Diseases: Molecular Pathophysiology and Novel Therapies

Rare Monogenic Diseases: Molecular Pathophysiology and Novel Therapies

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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
Published: 04/27/2023
Pages: 250
Binding Type: Hardcover
Weight: 1.63lbs
Size: 9.61h x 6.69w x 0.81d
ISBN: 9783036574615

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