Curative gene therapies for rare diseases - PMC
Diseases caused by alterations in the DNA can be overcome by providing the cells or tissues with a functional copy of the mutated gene. The most common form of gene therapy implies adding an extra genetic unit into the cell. However, new genome engineering ...
J Community Genet . 2020 Aug 15;12(2):267–276. doi: 10.1007/s12687-020-00480-6 Curative gene therapies for rare diseases Rocio Maldonado Rocio Maldonado 1 Stem Cells and Metabolism Research Program, University of Helsinki, Helsinki, Finland Find articles by Rocio Maldonado 1 , Sami Jalil Sami Jalil 1 Stem Cells and Metabolism Research Program, University of Helsinki, Helsinki, Finland Find articles by Sami Jalil 1 , Kirmo Wartiovaara Kirmo Wartiovaara 1 Stem Cells and Metabolism Research Program, University of Helsinki, Helsinki, Finland 2 Clinical Genetics, Helsinki University Hospital, Helsi
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