Treat Oculocutaneous Albinism with Gene Therapy

Abstract

Oculocutaneous albinism (OCA) is a group of hereditary recessive disorder recognized as a loss of pigmentation. OCA can derive from mutations in different genes that produce melanin. These mutations cause disturbances to get a standard melanin synthesis. There are 7 types of oculocutaneous albinism. These include OCA1, OCA2, OCA3, OCA4, OCA5, OCA6, and OCA7. To help OCA patients, it may include management of such as hats with brims and sunscreens. An effective therapy is unavailable for albinism at present. However, to fight OCA in the future, gene therapy can be used. Gene therapy can include use of such as retrovirus vectors, adenovirus vectors, and CRISPR/Cas9 system. Research results in animal models have shown remarkable advances. It means that the gene therapy will be helpful to treat people with albinism.

Keywords: albinism, albino, oculocutaneous albinism, gene therapy