Elivaldogene autotemcel

Elivaldogene autotemcel (pronunciation: eh-liv-al-doh-jeen aw-toh-tem-sel) is a gene therapy used for the treatment of Leber's congenital amaurosis (LCA), a rare genetic disorder affecting the retina at the back of the eye. It is the first gene therapy approved for a genetic disease in the United States and is sold under the brand name Luxturna.

Etymology

The name "Elivaldogene autotemcel" is derived from the International Nonproprietary Names (INN), where "Elivaldo" refers to the gene that is replaced (RPE65), "gene" signifies the gene therapy, "auto" indicates that the therapy is autologous (using the patient's own cells), and "temcel" is a stem cell.

Mechanism of Action

Elivaldogene autotemcel works by delivering a normal copy of the RPE65 gene directly into retinal cells. These cells then produce the normal protein that converts light to an electrical signal in the retina to restore patient's vision loss. Elivaldogene autotemcel uses a modified virus, known as a vector, to deliver the RPE65 gene to the retinal cells.

Related Terms

• Gene Therapy
• Leber's congenital amaurosis
• Retina
• RPE65
• Luxturna
• Vector

See Also

• Genetic Disorders
• Eye Diseases
• Retinal Diseases

External links

• Medical encyclopedia article on Elivaldogene autotemcel
• Wikipedia's article - Elivaldogene autotemcel

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