Chacón-Camacho ÓF, Zenteno JC

Language: Spanish
References: 6
Page: 276-278
PDF size: 65.66 Kb.

ABSTRACT

This is a significant time moment in the field of gene therapy in humans. Recently, results from a phase III clinical trial were published, demonstrating the first gene therapy success for a genetic disease. A clinical trial was carried out in patients suffering a hereditary blindness disease named Leber congenital amaurosis, caused by mutations in the RPE65 gene. Participating subjects received a subretinal injection of the normal RPE65 gene and one year after exhibited a significant improvement in visual acuity. It is expected that this gene therapy treatment will be approved by the FDA and commercialized in the USA in 2017.

REFERENCES

1. RetNet.2017. (Consultado en marzo de 2017) Disponible en: http:// www. sph.uth.tmc.edu.RetNet

2. Chacón-Camacho OF, Astorga-Carballo A, Zenteno JC. Terapia génica para enfermedades oftalmológicas: avances y perspectivas. Gac Med Mex. 2015;151:501-11.

3. Chung DC, Traboulsi EI. Leber congenital amaurosis: clinical correlations with genotypes, gene therapy trials update, and future directions. J AAPOS. 2009;13:587-92.

4. Chacón-Camacho OF, Zenteno JC. Review and update on the molecular basis of Leber congenital amaurosis. World J Clin Cases. 2015;16:112- 24.

5. Schimmer J, Breazzano S. Investor outlook: focus on upcoming LCA2 gene therapy phase III results. Hum Gene Ther Clin Dev. 2015;26:144-9.

6. Spark Therapeutics. (Consultado en marzo de 2017.) Disponible en: http://ir.sparktx.com/phoenix.zhtml?c=253900&p=irol-irhome