2020 Monogenic and Complex Diseases Gene therapy is a promising approach for both monogenic and complex diseases 1 1 Wang D Gau G Discov Med 201418151161 2 Ginn SL et al J Gene Med ID: 1045312
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1. Genetic Diseases and Gene Therapy Approaches2020
2. Monogenic and Complex DiseasesGene therapy is a promising approach for both monogenic and complex diseases11. Wang D, Gau G. Discov Med 2014;18:151–161; 2. Ginn SL, et al. J Gene Med 2018;20(5):e3015. Monogenic DiseasesMonogenic diseases are genetic disorders caused by modifications in a single gene occurring in all cells of the body1Examples include spinal muscular atrophy and inherited retinal disease2Complex DiseasesOther diseases have more complex roots, involving multiple genetic abnormalities in conjunction with other factors1Examples include cancer and heart failure1
3. Treatment Strategies for Monogenic DiseasesDifferent strategies are being utilised for the treatment of monogenic diseases. These include gene-based approaches as well as protein- and substrate-based therapies1,2Protein-based / protein-directed1,2RNA-based1DNA-based1Substrate-based2DNARNAmRNAmRNAAmino acidchainProteinNucleusCytoplasmFoldingTranslationExportSplicingTranscriptionSubstrateEnzyme1. Nature Education. Gene-based therapeutic approaches. Available at: https://www.nature.com/scitable/topicpage/gene-based-therapeutic-approaches-3881. Accessed 15 November 2018; 2. Gambello MJ, Li H. J Genet Genomics 2018;45(2):61–70.