Ontology highlight
ABSTRACT:
SUBMITTER: Damen M
PROVIDER: S-EPMC6148748 | biostudies-literature | 2018 Sep
REPOSITORIES: biostudies-literature
Damen M M Groenen A J J AJJ van Dongen S F M SFM Nolte R J M RJM Scholte B J BJ Feiters M C MC
MedChemComm 20180717 9
Diseases that are linked to defective genes or mutations can in principle be cured by gene therapy, in which damaged or absent genes are either repaired or replaced by new DNA in the nucleus of the cell. Related to this, disorders associated with elevated protein expression levels can be treated by RNA interference <i>via</i> the delivery of siRNA to the cytoplasm of cells. Polynucleotides can be brought into cells by viruses, but this is not without risk for the patient. Alternatively, DNA and ...[more]