scholarly journals Replacement Gene Therapy with a HumanRPGRIP1Sequence Slows Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis

2010 ◽  
Vol 21 (8) ◽  
pp. 993-1004 ◽  
Author(s):  
Basil S. Pawlyk ◽  
Oleg V. Bulgakov ◽  
Xiaoqing Liu ◽  
Xiaoyun Xu ◽  
Michael Adamian ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Murine Model ◽  
Photoreceptor Degeneration ◽  
Leber Congenital Amaurosis

scholarly journals Deletion of M-opsin prevents “M cone” degeneration in a mouse model of Leber congenital amaurosis

2019 ◽  
Author(s):  
Hui Xu ◽  
Nduka Enemchukwu ◽  
Xiaoyue Zhong ◽  
Olivia Zhang ◽  
Yingbin Fu
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
General Mechanism ◽  
Mouse Line ◽  
Photoreceptor Degeneration ◽  
Multiple Forms ◽  
Leber Congenital Amaurosis ◽  
Reporter Mouse ◽  
Therapy Strategy ◽  
Gene Therapy Strategy

AbstractMutations in RPE65 or lecithin-retinol acyltransferase (LRAT) disrupt 11-cis-retinal synthesis and cause Leber congenital amaurosis (LCA). In Lrat−/− mouse model, mislocalized medium (M)-wavelength sensitive opsin was degraded whereas mislocalized short (S)-wavelength sensitive opsin accumulated before the onset of cone degeneration. The mechanism for the foveal medium (M)/long (L)-wavelength-sensitive cone degeneration in LCA is unknown. By crossing Lrat−/− mice with a proteasome reporter mouse line, we showed that M-opsin enriched dorsal cones in Lrat−/− mice exhibit proteasome stress due to the degradation of large amounts of M-opsin. Deletion of M-opsin relieves the proteasome stress and completely prevents “M cone” degeneration in Lrat−/−Opn1sw−/− mice (a pure “M cone” LCA model, Opn1sw−/− encoding S-opsin) for at least 12 months. Our results suggest that M-opsin degradation associated proteasome stress plays a major role in “M cone” degeneration in Lrat−/− model. This finding may represent a general mechanism for “M cone” degeneration for multiple forms of cone degeneration due to M-opsin mislocalization and degradation. Our results have important implications for the current gene therapy strategy for LCA that emphasizes the need for a combinatorial therapy to both improve vision and slow photoreceptor degeneration.

scholarly journals Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis

2018 ◽  
Vol 29 (1) ◽  
pp. 42-50 ◽  
Author(s):  
Wei Zhang ◽  
Linjing Li ◽  
Qin Su ◽  
Guangping Gao ◽  
Hemant Khanna
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
Photoreceptor Degeneration ◽  
Leber Congenital Amaurosis

Gene Replacement Therapy Rescues Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis Lacking RPGRIP

2005 ◽  
Vol 46 (9) ◽  
pp. 3039 ◽  
Author(s):  
Basil S. Pawlyk ◽  
Alexander J. Smith ◽  
Prateek K. Buch ◽  
Michael Adamian ◽  
Dong-Hyun Hong ◽  
...  
Keyword(s):  
Replacement Therapy ◽  
Murine Model ◽  
Gene Replacement ◽  
Photoreceptor Degeneration ◽  
Leber Congenital Amaurosis ◽  
Gene Replacement Therapy

Gene Therapy for a Murine Model of Eosinophilic Esophagitis

Allergy ◽  
2021 ◽  
Author(s):  
Anna E. Camilleri ◽  
Saparja Nag ◽  
Anthony R. Russo ◽  
Katie M. Stiles ◽  
Ronald G. Crystal ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Eosinophilic Esophagitis ◽  
Murine Model

P0563 : RNAi-based gene therapy for chronic hepatitis B: Evaluation in a murine model

2015 ◽  
Vol 62 ◽  
pp. S526
Author(s):  
T. Michler ◽  
S. Grosse ◽  
S. Mockenhaupt ◽  
M. Heikenwaelder ◽  
D. Grimm ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Chronic Hepatitis ◽  
Hepatitis B ◽  
Chronic Hepatitis B ◽  
Murine Model

CaSm/gemcitabine chemo-gene therapy leads to prolonged survival in a murine model of pancreatic cancer

Surgery ◽  
2001 ◽  
Vol 130 (2) ◽  
pp. 280-288 ◽  
Author(s):  
Joseph R. Kelley ◽  
Melissa M. Fraser ◽  
Clifford W. Schweinfest ◽  
John N. Vournakis ◽  
Dennis K. Watson ◽  
...  
Keyword(s):  
Pancreatic Cancer ◽  
Gene Therapy ◽  
Murine Model ◽  
Prolonged Survival
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