Curative gene therapies for rare diseases.
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ABSTRACT:
SUBMITTER: Maldonado R
PROVIDER: S-EPMC8141081 | biostudies-literature | 2021 Apr
REPOSITORIES: biostudies-literature
Publications
Curative gene therapies for rare diseases.
Maldonado Rocio R Jalil Sami S Wartiovaara Kirmo K
Journal of community genetics 20200815 2
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 techniques also allow the modification or correction of the existing allele, providing new possibilities, especially for dominant diseases. Gene therapies have been tested for 30 years in thousands of clinical trials, but presently, we have on ...[more]
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