Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2017 Jan 27;8(2):53.
doi: 10.3390/genes8020053.

Correction of Monogenic and Common Retinal Disorders with Gene Therapy

Jesse D Sengillo  1   2   3 , Sally Justus  4   5 , Thiago Cabral  6   7   8   9 , Stephen H Tsang  10   11   12
Affiliations
Review

Correction of Monogenic and Common Retinal Disorders with Gene Therapy

Jesse D Sengillo et al. Genes (Basel). .

Abstract

The past decade has seen major advances in gene-based therapies, many of which show promise for translation to human disease. At the forefront of research in this field is ocular disease, as the eye lends itself to gene-based interventions due to its accessibility, relatively immune-privileged status, and ability to be non-invasively monitored. A landmark study in 2001 demonstrating successful gene therapy in a large-animal model for Leber congenital amaurosis set the stage for translation of these strategies from the bench to the bedside. Multiple clinical trials have since initiated for various retinal diseases, and further improvements in gene therapy techniques have engendered optimism for alleviating inherited blinding disorders. This article provides an overview of gene-based strategies for retinal disease, current clinical trials that engage these strategies, and the latest techniques in genome engineering, which could serve as the next frontline of therapeutic interventions.

Keywords: gene therapy, genome engineering, CRISPR, retinal degeneration, inherited retinal disease.

PubMed Disclaimer

Conflict of interest statement

The authors declare no conflicts of interest.

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. Raper S.E., Chirmule N., Lee F.S., Wivel N.A., Bagg A., Gao G.P., Wilson J.M., Batshaw M.L. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol. Genet. Metab. 2003;80:148–158. doi: 10.1016/j.ymgme.2003.08.016. - DOI - PubMed
    1. Kohn D.B., Sadelain M., Glorioso J.C. Occurrence of leukaemia following gene therapy of X-linked SCID. Nat. Rev. Cancer. 2003;3:477–488. doi: 10.1038/nrc1122. - DOI - PubMed
    1. Bennett J., Tanabe T., Sun D., Zeng Y., Kjeldbye H., Gouras P., Maguire A.M. Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy. Nat. Med. 1996;2:649–654. doi: 10.1038/nm0696-649. - DOI - PubMed
    1. Acland G.M., Aguirre G.D., Ray J., Zhang Q., Aleman T.S., Cideciyan A.V., Pearce-Kelling S.E., Anand V., Zeng Y., Maguire A.M., et al. Gene therapy restores vision in a canine model of childhood blindness. Nat. Genet. 2001;28:92–95. doi: 10.1038/ng0501-92. - DOI - PubMed
    1. Bainbridge J.W., Smith A.J., Barker S.S., Robbie S., Henderson R., Balaggan K., Viswanathan A., Holder G.E., Stockman A., Tyler N., et al. Effect of gene therapy on visual function in Leber’s congenital amaurosis. N. Engl. J. Med. 2008;358:2231–2239. doi: 10.1056/NEJMoa0802268. - DOI - PubMed