scholarly journals Long-term Retinal Function and Structure Rescue Using Capsid Mutant AAV8 Vector in the rd10 Mouse, a Model of Recessive Retinitis Pigmentosa

2011 ◽  
Vol 19 (2) ◽  
pp. 234-242 ◽  
Author(s):  
Ji-jing Pang ◽  
Xufeng Dai ◽  
Shannon E Boye ◽  
Ilaria Barone ◽  
Sanford L Boye ◽  
...  
Keyword(s):  
Retinitis Pigmentosa ◽  
Retinal Function

scholarly journals Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy

Gene Therapy ◽  
2005 ◽  
Vol 12 (8) ◽  
pp. 694-701 ◽  
Author(s):  
M Tschernutter ◽  
F C Schlichtenbrede ◽  
S Howe ◽  
K S Balaggan ◽  
P M Munro ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Retinitis Pigmentosa ◽  
Rat Model ◽  
Retinal Function ◽  
Rcs Rat ◽  
Long Term Preservation

Normal Central Retinal Function and Structure Preserved in Retinitis Pigmentosa

2010 ◽  
Vol 51 (2) ◽  
pp. 1079 ◽  
Author(s):  
Samuel G. Jacobson ◽  
Alejandro J. Roman ◽  
Tomas S. Aleman ◽  
Alexander Sumaroka ◽  
Waldo Herrera ◽  
...  
Keyword(s):  
Retinitis Pigmentosa ◽  
Retinal Function

scholarly journals Beyond Sector Retinitis Pigmentosa: Expanding the Phenotype and Natural History of the Rhodopsin Gene Codon 106 Mutation (Gly-to-Arg) in Autosomal Dominant Retinitis Pigmentosa

Genes ◽  
2021 ◽  
Vol 12 (12) ◽  
pp. 1853
Author(s):  
Brian G. Ballios ◽  
Emily M. Place ◽  
Luis Martinez-Velazquez ◽  
Eric A. Pierce ◽  
Jason I. Comander ◽  
...  
Keyword(s):  
Retinitis Pigmentosa ◽  
Autosomal Dominant ◽  
Peripheral Vision ◽  
Visual Fields ◽  
Macular Disease ◽  
History Of ◽  
Intrafamilial Variability ◽  
Long Term Prognosis

Sector and pericentral are two rare, regional forms of retinitis pigmentosa (RP). While usually defined as stable or only very slowly progressing, the available literature to support this claim is limited. Additionally, few studies have analyzed the spectrum of disease within a particular genotype. We identified all cases (9 patients) with an autosomal dominant Rhodopsin variant previously associated with sector RP (RHO c.316G > A, p.Gly106Arg) at our institution. Clinical histories were reviewed, and testing included visual fields, multimodal imaging, and electroretinography. Patients demonstrated a broad phenotypic spectrum that spanned regional phenotypes from sector-like to pericentral RP, as well as generalized disease. We also present evidence of significant intrafamilial variability in regional phenotypes. Finally, we present the longest-reported follow-up for a patient with RHO-associated sector-like RP, showing progression from sectoral to pericentral disease over three decades. In the absence of comorbid macular disease, the long-term prognosis for central visual acuity is good. However, we found that significant progression of RHO p.Gly106Arg disease can occur over protracted periods, with impact on peripheral vision. Longitudinal widefield imaging and periodic ERG reassessment are likely to aid in monitoring disease progression.

Long-term Outcomes of Cataract Surgery in Patients with Retinitis Pigmentosa

2021 ◽  
Author(s):  
Shun Nakamura ◽  
Kohta Fujiwara ◽  
Noriko Yoshida ◽  
Yusuke Murakami ◽  
Shotaro Shimokawa ◽  
...  
Keyword(s):  
Retinitis Pigmentosa ◽  
Cataract Surgery ◽  
Long Term Outcomes

Retinal blood flow velocity measured by retinal function imaging in retinitis pigmentosa

2011 ◽  
Vol 249 (12) ◽  
pp. 1855-1858 ◽  
Author(s):  
Sven C. Beutelspacher ◽  
Nermin Serbecic ◽  
Hila Barash ◽  
Zvia Burgansky-Eliash ◽  
Amiram Grinvald ◽  
...  
Keyword(s):  
Blood Flow ◽  
Retinitis Pigmentosa ◽  
Flow Velocity ◽  
Blood Flow Velocity ◽  
Retinal Function ◽  
Retinal Blood Flow ◽  
Retinal Blood Flow Velocity

Long-term follow-up for efficacy and safety of treatment of retinitis pigmentosa with valproic acid

2013 ◽  
Vol 97 (7) ◽  
pp. 895-899 ◽  
Author(s):  
Sheena Bhalla ◽  
Deval Joshi ◽  
Shaminder Bhullar ◽  
Daniel Kasuga ◽  
Yeonhee Park ◽  
...  
Keyword(s):  
Valproic Acid ◽  
Retinitis Pigmentosa ◽  
Efficacy And Safety ◽  
Long Term Follow Up

scholarly journals Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease

2015 ◽  
Vol 112 (43) ◽  
pp. E5844-E5853 ◽  
Author(s):  
William A. Beltran ◽  
Artur V. Cideciyan ◽  
Simone Iwabe ◽  
Malgorzata Swider ◽  
Mychajlo S. Kosyk ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Retinitis Pigmentosa ◽  
Large Animal Model ◽  
Therapeutic Window ◽  
Large Animal ◽  
Visual Behavior ◽  
Rate Of Progression ◽  
Photoreceptor Neurons ◽  
Retinal Gene Therapy

Inherited retinal degenerations cause progressive loss of photoreceptor neurons with eventual blindness. Corrective or neuroprotective gene therapies under development could be delivered at a predegeneration stage to prevent the onset of disease, as well as at intermediate-degeneration stages to slow the rate of progression. Most preclinical gene therapy successes to date have been as predegeneration interventions. In many animal models, as well as in human studies, to date, retinal gene therapy administered well after the onset of degeneration was not able to modify the rate of progression even when successfully reversing dysfunction. We evaluated consequences of gene therapy delivered at intermediate stages of disease in a canine model of X-linked retinitis pigmentosa (XLRP) caused by a mutation in the Retinitis Pigmentosa GTPase Regulator (RPGR) gene. Spatiotemporal natural history of disease was defined and therapeutic dose selected based on predegeneration results. Then interventions were timed at earlier and later phases of intermediate-stage disease, and photoreceptor degeneration monitored with noninvasive imaging, electrophysiological function, and visual behavior for more than 2 y. All parameters showed substantial and significant arrest of the progressive time course of disease with treatment, which resulted in long-term improved retinal function and visual behavior compared with control eyes. Histology confirmed that the humanRPGRtransgene was stably expressed in photoreceptors and associated with improved structural preservation of rods, cones, and ON bipolar cells together with correction of opsin mislocalization. These findings in a clinically relevant large animal model demonstrate the long-term efficacy ofRPGRgene augmentation and substantially broaden the therapeutic window for intervention in patients withRPGR-XLRP.

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