Glosario de términos

Off-target effects occur when the genome editing tool, such as CRISPR-Cas9 system, cuts DNA at unintended locations in the genome. This can potentially cause unwanted genetic modifications, such as insertions or deletions at the wrong site.

An oncogene is a gene that, when mutated, or expressed at abnormal levels, has the potential to cause cancer. It does this by promoting abnormal cell growth.

A designation given to medicines during development which allows the sponsor to access incentives to encourage the development of medicines for rare diseases. This designation applies specifically to medicines for very rare, life-threatening or debilitating diseases, for which there is no satisfactory treatment available or for which the new medicine would provide a significant benefit.