scholarly journals Genetic Reactivation of Cone Photoreceptors Restores Visual Responses in Retinitis Pigmentosa

Science ◽  
2010 ◽  
Vol 329 (5990) ◽  
pp. 413-417 ◽  
Author(s):  
V. Busskamp ◽  
J. Duebel ◽  
D. Balya ◽  
M. Fradot ◽  
T. J. Viney ◽  
...  
2011 ◽  
Vol 11 (1) ◽  
Author(s):  
Emmanuelle Clérin ◽  
Nicolas Wicker ◽  
Saddek Mohand-Saïd ◽  
Olivier Poch ◽  
José-Alain Sahel ◽  
...  

2009 ◽  
Vol 12 (1) ◽  
pp. 5-6 ◽  
Author(s):  
Paola Bovolenta ◽  
Elsa Cisneros

2021 ◽  
Author(s):  
Yunlu Xue ◽  
Sean K. Wang ◽  
Parimal Rana ◽  
Emma R. West ◽  
Christin M. Hong ◽  
...  

AbstractRetinitis pigmentosa (RP) is an inherited retinal disease, affecting >20 million people worldwide. Loss of daylight vision typically occurs due to the dysfunction/loss of cone photoreceptors, the cell type that initiates our color and high acuity vision. Currently, there is no effective treatment for RP, other than gene therapy for a limited number of specific disease genes. To develop a gene-agnostic therapy, we screened ≈20 genes for their ability to prolong cone photoreceptor survival in vivo. Here, we report an adeno-associated virus (AAV) vector expressing Txnip, which prolongs the survival of cone photoreceptors and improves visual acuity in RP mouse models. A Txnip allele, C247S, which blocks the association of Txnip with thioredoxin, provides an even greater benefit. Additionally, the rescue effect of Txnip depends on lactate dehydrogenase b (Ldhb), and correlates with the presence of healthier mitochondria, suggesting that Txnip saves RP cones by enhancing their lactate catabolism.


Author(s):  
Ayoub Lassoued ◽  
Furu Zhang ◽  
Kazuhiro Kurokawa ◽  
Yan Liu ◽  
James A. Crowell ◽  
...  

2013 ◽  
Vol 251 (7) ◽  
pp. 1669-1677 ◽  
Author(s):  
José-Alain Sahel ◽  
Thierry Léveillard ◽  
Serge Picaud ◽  
Deniz Dalkara ◽  
Katia Marazova ◽  
...  

2021 ◽  
Vol 0 (0) ◽  
pp. 0
Author(s):  
HenryJ Kaplan ◽  
Wei Wang ◽  
Niloofar Piri ◽  
DouglasC Dean

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