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 mechani...
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Format: | Electronic Book Chapter |
Language: | English |
Published: |
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MDPI - Multidisciplinary Digital Publishing Institute
2023
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Online Access: | DOAB: download the publication DOAB: description of the publication |
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Summary: | 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. |
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Physical Description: | 1 electronic resource (250 p.) |
ISBN: | books978-3-0365-7460-8 9783036574615 9783036574608 |
Access: | Open Access |