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.
journal_name
Adv Exp Med Bioljournal_title
Advances in experimental medicine and biologyauthors
LaVail MM,Yasumura D,Matthes MT,Yang H,Hauswirth WW,Deng WT,Vollrath Ddoi
10.1007/978-3-319-17121-0_65subject
Has Abstractpub_date
2016-01-01 00:00:00pages
487-93eissn
0065-2598issn
2214-8019journal_volume
854pub_type
杂志文章abstract：:Brain lateralization is a ubiquitous feature of neural organization across the vertebrate spectrum. We have developed a model of motor lateralization that attributes different motor control processes to each cerebral hemisphere. This bilateral hemispheric model of motor control has successfully predicted hemisphere-sp...
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journal_title：Advances in experimental medicine and biology
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journal_title：Advances in experimental medicine and biology
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journal_title：Advances in experimental medicine and biology
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