Gene therapy is a platform of technologies used to develop treatments for genetic disorders and other diseases.
For diseases that are caused by a single gene, gene replacement therapy can treat the disease by replacing the missing or defective gene with a healthy functioning copy.
Genome editing (or gene editing) is the precise manipulation of an organism’s DNA sequence using engineered nucleases to insert, delete, or replace nucleotides at specific locations within a genome. For therapeutic applications, this approach could be used to correct a gene mutation and restore function or to inactivate a harmful mutant gene. Nuclease-based gene editing technologies include meganucleases, zinc finger nucleases, TALENs, and most recently CRISPR-Cas9 and CRISPR-Cpf1.
Monoclonal antibodies (mAbs) can be delivered to specific cells or tissues. mAbs work by binding to a target cell and inducing the immune system to attack that cell. To date, mAbs have been used to treat infectious diseases, autoimmune disorders, metabolic disorders, and cancer.
Gene silencing therapy, also known as RNA interference, interrupts gene expression at the intermediate step between DNA and protein by targeting messenger RNA (mRNA) before it is translated into protein. Some genetic diseases result in the overproduction of “bad” proteins, so reducing or eliminating the amount of a faulty protein that is expressed can be therapeutic. Small interfering RNAs (siRNAs) and antisense oligonucleotides (ASOs) can be designed to pair with a specific mRNA strand and causes it to be degraded.