Nonsense Mutation Correction in Human Diseases An Approach for Targeted Medicine

Nonsense Mutation Correction in Human DiseasesAn Approach for Targeted Medicine\nAuthor(s): Fabrice Lejeune, Hana Benhabiles, Jieshuang Jia\nFormat: Paperback\nPublisher: Elsevier Science Publishing Co Inc, United States\nImprint: Academic Press Inc\nISBN-13: 9780128044681, 978-0128044681\nSynopsis\nNonsense Mutation Correction in Human Diseases: An Approach for Targeted Medicine provides an introduction on genetic diseases, discusses the prevalence of nonsense mutations, the consequences of a nonsense mutation for the expression of the mutant gene, and the presentation of the nonsense-mediated mRNA decay (NMD). It presents the mechanism of action and rationale associated with each strategy to correct nonsense mutations with the results of clinical trials to further support this basis. In addition, the book shows how it may be possible to combine several of these strategies to ultimately improve the efficiency of correction, also suggesting the future goals and objectives to improve.