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Gene Therapy for MERTK-Associated Retinal Degenerations.

Gene Therapy for MERTK-Associated Retinal Degenerations.

Authors :
LaVail MM
Yasumura D
Matthes MT
Yang H
Hauswirth WW
Deng WT
Vollrath D
Source :
Advances in experimental medicine and biology [Adv Exp Med Biol] 2016; Vol. 854, pp. 487-93.
Publication Year :
2016

Abstract

MERTK-associated retinal degenerations are thought to have defects in phagocytosis of shed outer segment membranes by the retinal pigment epithelium (RPE), as do the rodent models of these diseases. We have subretinally injected an RPE-specific AAV2 vector, AAV2-VMD2-hMERTK, to determine whether this would provide long-term photoreceptor rescue in the RCS rat, which it did for up to 6.5 months, the longest time point examined. Moreover, we found phagosomes in the RPE in the rescued regions of RCS retinas soon after the onset of light. The same vector also had a major protective effect in Mertk-null mice, with a concomitant increase in ERG response amplitudes in the vector-injected eyes. These findings suggest that planned clinical trials with this vector will have a favorable outcome.

Details

Language :
English
ISSN :
0065-2598
Volume :
854
Database :
MEDLINE
Journal :
Advances in experimental medicine and biology
Publication Type :
Academic Journal
Accession number :
26427450
Full Text :
https://doi.org/10.1007/978-3-319-17121-0_65