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
. 2015 Mar:12:52-70.
Epub 2015 Mar 1.

Emerging Treatments for Retinitis Pigmentosa: Genes and stem cells, as well as new electronic and medical therapies, are gaining ground

Michael K Lin  1 , Yi-Ting Tsai  1 , Stephen H Tsang  1
Affiliations

Emerging Treatments for Retinitis Pigmentosa: Genes and stem cells, as well as new electronic and medical therapies, are gaining ground

Michael K Lin et al. Retin Physician. 2015 Mar.
No abstract available

PubMed Disclaimer

Conflict of interest statement

None of the authors reports any financial interest in any of the products mentioned in this article.

Figures

Figure 1
Figure 1
Schematic illustration of gene therapy and gene repair. A) Diseases caused by recessive gene mutations can be treated by gene supplementation therapy. B) In diseases caused by dominant gene mutations, however, the protein produced by the mutated gene destroys the function of normal gene products, preventing gene supplementation therapy from being effective. Correcting the mutation with gene repair can treat the disease.
Figure 2
Figure 2
Fundus photograph from a six-year-old with early-onset retinal dystrophy caused by mutation in the gene encoding RPE65 (K295X and H68Y). Classic salt-and-pepper mottling can be seen.
Figure 3
Figure 3
Retinal photograph of a 60-electrode Argus II retinal prosthesis.
See this image and copyright information in PMC

References

    1. Berson EL, Rosner B, Sandberg MA, et al. A randomized trial of vitamin A and vitamin E supplementation for retinitis pigmentosa. Arch Ophthalmol. 1993;111:761–772. - PubMed
    1. Berson EL, Rosner B, Sandberg MA, et al. Further evaluation of docosahexaenoic acid in patients with retinitis pigmentosa receiving vitamin A treatment: subgroup analyses. Arch Ophthalmol. 2004;122:1306–1314. - PubMed
    1. Bainbridge JWB, Smith AJ, Barker SS, et al. Effect of gene therapy on visual function in Leber’s congenital amaurosis. N Engl J Med. 2008;358:2231–2239. - PubMed
    1. Hauswirth WW, Aleman TS, Kaushal S, et al. Treatment of Leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther. 2008;19:979–990. - PMC - PubMed
    1. Maguire AM, Simonelli F, Pierce EA, et al. Safety and Efficacy of gene transfer for Leber’s congenital amaurosis. N Engl J Med. 2008;358:2240–2248. - PMC - PubMed

LinkOut - more resources