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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Detalles Bibliográficos
Otros Autores: Condò, Ivano, editor (editor)
Formato: Libro electrónico
Idioma:Inglés
Publicado: [Place of publication not identified] : MDPI - Multidisciplinary Digital Publishing Institute 2023.
Materias:
Ver en Biblioteca Universitat Ramon Llull:https://discovery.url.edu/permalink/34CSUC_URL/1im36ta/alma991009741080206719
Descripción
Sumario: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.
Descripción Física:1 online resource (250 pages)
ISBN:9783036574608