Format

Send to

Choose Destination
See comment in PubMed Commons below
Curr Gene Ther. 2017 Nov 29. doi: 10.2174/1566523218666171129204926. [Epub ahead of print]

The Progress of Gene Therapy for Leber`s Optic Hereditary Neuropathy.

Author information

1
Taihe Hospital, Hubei University of Medicine - Department of Ophthalmology Shiyan, Hubei. China.
2
Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology - Department of Ophthalmology Wuhan, Hubei. China.
3
Taihe Hospital, Hubei University of Medicine - Department of Ophthalmology Shiyan, Hubei,China Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology - Department of Ophthalmology Wuhan, Hubei. China.

Abstract

Leber's Optic Hereditary Neuropathy (LHON) is a common cause of teenaged blindness in both eyes for which there is currently no effective treatment. In 1871, the German ophthalmologist Theodor Leber was the first to describe the clinical characteristics of his namesake disease, and through unremitting efforts over the past 100 years, researchers have continued to increase their understanding of LHON. In recent years, using gene therapy, several groups have obtained breakthroughs in the treatment of the disease. In this article, we will review the challenging journey that researchers faced towards our current understanding of LHON, and describe the transition of gene therapy research for LHON from the bench to bedside.

KEYWORDS:

Allotopic Expression Technology.; Gene Therapy; Leber's Optic Hereditary Neuropathy; MT-ND4; Mitochondrial Disease; Translational Medicine

PubMed Commons home

PubMed Commons

0 comments
How to join PubMed Commons

    Supplemental Content

    Full text links

    Icon for Bentham Science Publishers Ltd.
    Loading ...
    Support Center