Is Exondys 51 a type of gene therapy?

Drugs.com

Official answer

by Drugs.com

Exondys 51 is not technically gene therapy, but it does treat people with Duchenne muscular dystrophy who have a confirmed genetic mutation in their dystrophin gene which means they are unable to make the protein dystrophin. Dystrophin is needed for muscle repair. Without it, muscle cells become damaged and weakened over time, which can affect voluntary movement, such as walking.

Gene therapy technically refers to any technique that modifies a person's genes to treat or cure disease. The therapy may involve replacing a disease-causing gene with a healthy copy or inactivating a disease-causing gene that does not work properly. Exondys 51 does not work like this. Instead, it skips exon 51 (exons are sequences of DNA present in mature messenger RNA, some of which encode for specific proteins), which allows the body to make a shorter form of the dystrophin protein which still has some function. Exondys 51 is not a cure for muscular dystrophy but may lessen muscle weakness and muscle wasting. But it can only be used by children with a confirmed genetic mutation in their dystrophin gene that responds to skipping exon 51.

Exondys 51 belongs to the class of medicines known as antisense oligonucleotides. These help cells skip over a specific exon during splicing, which allows them to join a different set of exons together to produce a protein that is shorter than usual but may have some function.

Related medical questions

Disclaimer

Every effort has been made to ensure that the information provided by Drugslib.com is accurate, up-to-date, and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. Drugslib.com information has been compiled for use by healthcare practitioners and consumers in the United States and therefore Drugslib.com does not warrant that uses outside of the United States are appropriate, unless specifically indicated otherwise. Drugslib.com's drug information does not endorse drugs, diagnose patients or recommend therapy. Drugslib.com's drug information is an informational resource designed to assist licensed healthcare practitioners in caring for their patients and/or to serve consumers viewing this service as a supplement to, and not a substitute for, the expertise, skill, knowledge and judgment of healthcare practitioners.

The absence of a warning for a given drug or drug combination in no way should be construed to indicate that the drug or drug combination is safe, effective or appropriate for any given patient. Drugslib.com does not assume any responsibility for any aspect of healthcare administered with the aid of information Drugslib.com provides. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the drugs you are taking, check with your doctor, nurse or pharmacist.

Popular Keywords