Evidence map›Paper›PMID 26427450›Full record

ArticleAdvances in experimental medicine and biology2016

Gene Therapy for MERTK-Associated Retinal Degenerations.

Matthew M LaVail, Douglas Yasumura, Michael T Matthes, Haidong Yang, William W Hauswirth, Wen-Tao Deng, Douglas Vollrath

Abstract read
In one paragraph

Article in Advances in experimental medicine and biology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.

0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

21 citing papers in PubMed.

  1. Article
  2. Review
  3. Article
  4. Cell-cell interaction in the pathogenesis of inherited retinal diseases.Frontiers in cell and developmental biology · 2024
    Review
  5. Regulation of the rhythmic diversity of daily photoreceptor outer segment phagocytosis in vivo.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2022
    Review
  6. Review
  7. The LratInternational journal of molecular sciences · 2021
    Article
  8. Article
  9. Annals of translational medicine · 2020
    Review
  10. Review
  11. Gene Therapy in Retinal Dystrophies.International journal of molecular sciences · 2019
    Review
  12. Article
  13. Article
  14. Article
  15. Review
  16. Review
  17. Article
  18. Article
  19. Article
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors.

Matthew M LaVailBeckman Vision Center, UCSF School of Medicine, 10 Koret Way, 94143-0730, San Francisco, CA, USA. matthew.lavail@ucsf.edu.
Douglas YasumuraBeckman Vision Center, UCSF School of Medicine, 10 Koret Way, 94143-0730, San Francisco, CA, USA. matthew.lavail@ucsf.edu.
Michael T MatthesBeckman Vision Center, UCSF School of Medicine, 10 Koret Way, 94143-0730, San Francisco, CA, USA. Michael.Matthes@ucsf.edu.
Haidong YangBeckman Vision Center, UCSF School of Medicine, 10 Koret Way, 94143-0730, San Francisco, CA, USA. yang.harvey@gmail.com.
William W HauswirthDepartment of Ophthalmology, College of Medicine, University of Florida, 32610-0284, Gainesville, FL, USA. hauswrth@ufl.edu.
Wen-Tao DengDepartment of Ophthalmology, College of Medicine, University of Florida, 32610-0284, Gainesville, FL, USA.
Douglas VollrathDepartment of Genetics, Stanford University School of Medicine, 94305, Stanford, CA, USA. Vollrath@stanford.edu.

Funding

Rapid-Prototyping and Design CoreP30EY002162 · NEI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI OU, YVONNE · 1985 to 2024
$16.8M
CELLULAR MECHANISMS OF INHERITED RETINAL DEGENERATIONR01EY001919 · NEI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI LAVAIL, MATTHEW M · 1985 to 2014
$8.3M
RODENT MODELS OF RETINAL DEGENERATIONR01EY006842 · NEI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI LAVAIL, MATTHEW M · 1992 to 2014
$3.6M
NEUROPHYSIOLOGY OF INTERMEDIATE LEVEL VISIONF32EY006842 · NEI · ROCKEFELLER UNIVERSITY · PI BAKIN, JONATHAN S · 1997 to 1999
–
CELLULAR MECHANISMS OF INHERITED RETINAL DEGENERATIONR37EY001919 · NEI · UNIVERSITY OF CALIFORNIA SAN FRANCISCO · PI LAVAIL, MATTHEW M · 1989 to 1998
–
NEI NIH HHS EY001919NEI NIH HHS EY002162NEI NIH HHS EY006482NEI NIH HHS F32 EY006842NEI NIH HHS P30 EY002162NEI NIH HHS R01 EY001919NEI NIH HHS R01 EY006842
6 · The paper itself

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.

Indexed as

AnimalsBestrophinsChloride Channelsc-Mer Tyrosine KinaseDependovirusDisease Models, AnimalElectroretinographyEye ProteinsGenetic TherapyGenetic VectorsHumansMice, KnockoutPhagocytosisPhagosomesPromoter Regions, GeneticProto-Oncogene ProteinsBEST1 protein, humanBestrophinsChloride Channelsc-Mer Tyrosine KinaseEye ProteinsMERTK protein, humanMertk protein, mouseMertk protein, ratProto-Oncogene ProteinsReceptor Protein-Tyrosine KinasesGene therapyMERTKPhagocytosisRetinal degenerationTreatment

Identifiers

PMID26427450
PMCPMC4942279

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.