Question

Diseases caused by single-gene defects, such as cystic fibrosis, hemophilia, muscular

Answer 1

Answer:

Gene therapy

Explanation:

Gene therapy can be defined as the methodologies used to modify/restore the expression of target genes and thus cure and treat genetic disorders. Gene therapy approaches consist of delivering specific nucleic acids (either RNA or DNA) in the cells of the individual to be treated. Some of the most important gene therapy approaches include the use of small interfering RNAs (siRNAs) in order to block target gene expression, adenovirus vectors to insert genes into host cells, the use of the CRISPR-Cas9 genome editing system to insert specific sequences by the mechanism of homologous recombination, etc.