scholarly journals Gene therapy into photoreceptors and Müller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse models

2015 ◽  
Vol 24 (11) ◽  
pp. 3104-3118 ◽  
Author(s):  
Lucie P. Pellissier ◽  
Peter M. Quinn ◽  
C. Henrique Alves ◽  
Rogier M. Vos ◽  
Jan Klooster ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Retinitis Pigmentosa ◽  
Mouse Models ◽  
Glial Cells ◽  
Structure And Function ◽  
Müller Glial Cells ◽  
Retinal Structure ◽  
And Function

scholarly journals Direct comparison of retinal structure and function in retinitis pigmentosa by co-registering microperimetry and optical coherence tomography

PLoS ONE ◽  
2019 ◽  
Vol 14 (12) ◽  
pp. e0226097 ◽  
Author(s):  
Jun Funatsu ◽  
Yusuke Murakami ◽  
Shunji Nakatake ◽  
Masato Akiyama ◽  
Kohta Fujiwara ◽  
...  
Keyword(s):  
Optical Coherence Tomography ◽  
Retinitis Pigmentosa ◽  
Structure And Function ◽  
Optical Coherence ◽  
Retinal Structure ◽  
And Function

scholarly journals Quantitative Analysis of Retinal Structure and Function in Two Chromosomally Altered Mouse Models of Down Syndrome

2020 ◽  
Vol 61 (5) ◽  
pp. 25
Author(s):  
Daniella B. Victorino ◽  
Jonah J. Scott-McKean ◽  
Mark W. Johnson ◽  
Alberto C. S. Costa
Keyword(s):  
Down Syndrome ◽  
Quantitative Analysis ◽  
Mouse Models ◽  
Structure And Function ◽  
Retinal Structure ◽  
And Function

scholarly journals Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration

2020 ◽  
Vol 18 ◽  
pp. 582-594
Author(s):  
Scott H. Greenwald ◽  
Emily E. Brown ◽  
Michael J. Scandura ◽  
Erin Hennessey ◽  
Raymond Farmer ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
Retinal Degeneration ◽  
Structure And Function ◽  
Retinal Structure ◽  
And Function

scholarly journals Soluble CX3CL1 gene therapy improves cone survival and function in mouse models of retinitis pigmentosa

2019 ◽  
Vol 116 (20) ◽  
pp. 10140-10149 ◽  
Author(s):  
Sean K. Wang ◽  
Yunlu Xue ◽  
Parimal Rana ◽  
Christin M. Hong ◽  
Constance L. Cepko
Keyword(s):  
Gene Therapy ◽  
Retinitis Pigmentosa ◽  
Mouse Models ◽  
Normal Numbers ◽  
Cytokine Levels ◽  
Genes Encoding ◽  
High Acuity ◽  
Transcriptional Changes ◽  
And Function ◽  
Subretinal Injection

Retinitis pigmentosa (RP) is a disease that initially presents as night blindness due to genetic deficits in the rod photoreceptors of the retina. Rods then die, causing dysfunction and death of cone photoreceptors, the cell type that mediates high acuity and color vision, ultimately leading to blindness. We investigated immune responses in mouse models of RP and found evidence of microglia activation throughout the period of cone degeneration. Using adeno-associated vectors (AAVs), delivery of genes encoding microglial regulatory signals led to the identification of AAV serotype 8 (AAV8) soluble CX3CL1 (sCX3CL1) as a promising therapy for degenerating cones. Subretinal injection of AAV8-sCX3CL1 significantly prolonged cone survival in three strains of RP mice. Rescue of cones was accompanied by improvements in visual function. AAV8-sCX3CL1 did not affect rod survival, microglia localization, or inflammatory cytokine levels in the retina. Furthermore, although RNA sequencing of microglia demonstrated marked transcriptional changes with AAV8-sCX3CL1, pharmacological depletion of up to ∼99% of microglia failed to abrogate the effect of AAV8-sCX3CL1 on cone survival. These findings indicate that AAV8-sCX3CL1 can rescue cones in multiple mouse models of RP via a pathway that does not require normal numbers of microglia. Gene therapy with sCX3CL1 is a promising mutation-independent approach to preserve vision in RP and potentially other forms of retinal degeneration.

scholarly journals EYS Mutations Causing Autosomal Recessive Retinitis Pigmentosa: Changes of Retinal Structure and Function with Disease Progression

Genes ◽  
2017 ◽  
Vol 8 (7) ◽  
pp. 178 ◽  
Author(s):  
David McGuigan ◽  
Elise Heon ◽  
Artur Cideciyan ◽  
Rinki Ratnapriya ◽  
Monica Lu ◽  
...  
Keyword(s):  
Retinitis Pigmentosa ◽  
Disease Progression ◽  
Autosomal Recessive ◽  
Structure And Function ◽  
Retinal Structure ◽  
And Function
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