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https://www.readbyqxmd.com/read/29210653/human-umbilical-cord-mesenchymal-stem-cells-subpopulations-and-their-difference-in-cell-biology-and-effects-on-retinal-degeneration-in-rcs-rats
#1
L Wang, P Li, Y Tian, Z Li, C Lian, Q Ou, C Jin, F Gao, J-Y Xu, J Wang, F Wang, J Zhang, J Zhang, W Li, H Tian, L Lu, G-T Xu
Human umbilical cord mesenchymal stem cells (hUC-MSCs) are potential candidates for treating retinal degeneration (RD). To further study the biology and therapeutic effects of these cells, two hUC-MSC subpopulations, termed hUC-MSC1 and hUC-MSC2, were isolated by single-cell cloning method and their therapeutic functions were compared in RCS rat, a RD model. Although both subsets satisfied the basic requirements for hUC-MSCs, they were significantly different in morphology, proliferation rate, differentiation capacity, phenotype and gene expression...
December 5, 2017: Current Molecular Medicine
https://www.readbyqxmd.com/read/29160102/improving-the-infection-of-bone-marrow-derived-cells-with-an-integrase-defective-lentiviral-vector
#2
S Louise Pay, Xiaoping Qi, Jeffrey F Willard, Juliana Godoy, Kavya Sankhavaram, Ranier Horton, Sayak K Mitter, Judith Quigley, Chang Lung-Ji, Maria B Grant, Michael E Boulton
In lentiviral vector (LV) applications where transient transgene expression is sufficient, integrase-defective lentiviral vectors (IDLVs) are beneficial for reducing the potential for off-target effects associated with insertional mutagenesis. We previously demonstrated that human RPE65 mRNA expression from an integrating lentiviral vector (ILV) induces endogenous Rpe65 and Cralbp mRNA expression in murine bone marrow-derived cells (BMDCs), initiating programming of the cells to retinal pigment epithelium (RPE)-like cells...
November 21, 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/29133760/a-gene-scan-study-of-rpe65-in-chinese-patients-with-leber-congenital-amaurosis
#3
Jing Liu, Juan Bu
BACKGROUND: Leber congenital amaurosis (LCA) is a visual disease which is caused by RPE65 mutations and results in retinal degeneration and severe vision loss in early infancy. According to previous researches, mutations of the RPE65 gene account for 16% of all cases of LCA. This study aimed to identify RPE65 gene mutations in Chinese patients with LCA. METHODS: We recruited 52 sporadic patients from Peking University Third Hospital in 2016 and applied Sanger sequencing to identify variants among exons responsible for the disease...
November 20, 2017: Chinese Medical Journal
https://www.readbyqxmd.com/read/29097191/molecular-genetics-and-emerging-therapies-for-retinitis-pigmentosa-basic-research-and-clinical-perspectives
#4
REVIEW
Marina França Dias, Kwangsic Joo, Jessica A Kemp, Silvia Ligório Fialho, Armando da Silva Cunha, Se Joon Woo, Young Jik Kwon
Retinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people worldwide. It is characterized with progressive loss of rods and cones and causes severe visual dysfunction and eventual blindness in bilateral eyes. In addition to more than 3000 genetic mutations from about 70 genes, a wide genetic overlap with other types of retinal dystrophies has been reported with RP. This diversity of genetic pathophysiology makes treatment extremely challenging. Although therapeutic attempts have been made using various pharmacologic agents (neurotrophic factors, antioxidants, and anti-apoptotic agents), most are not targeted to the fundamental cause of RP, and their clinical efficacy has not been clearly proven...
October 31, 2017: Progress in Retinal and Eye Research
https://www.readbyqxmd.com/read/29057462/igf-1-activates-pi3k-akt-signaling-to-protect-human-retinal-pigment-epithelial-cells-from-amiodarone-induced-oxidative-injury
#5
Rifang Liao, Fengxia Yan, Zhuanping Zeng, Haitao Wang, Kaifeng Qiu, Jinying Xu, Wenhua Zheng
BACKGROUND AND PURPOSE: Amiodarone (AM) is one of the most effective anti-arrhythmic drugs available. However, clinical applications of AM are limited by its toxic side effects including optic toxicity. The purpose of this study was to investigate the toxic effect of AM on D407 cells (a human Retinal Pigmented Epithelial cell line) and the mechanisms of the protective effect of insulin-like growth factor-1 (IGF-1). EXPERIMENTAL APPROACH: The involvement of the kinases, Akt and Erk, was analyzed by Western blot...
October 22, 2017: British Journal of Pharmacology
https://www.readbyqxmd.com/read/29042966/effects-of-rpe-conditioned-medium-on-the-differentiation-of-hadscs-into-rpe-cells-and-their-proliferation-and-migration
#6
Yi Zhang, Dandan Zhang, Wei Wei, Bingqiao Shen, Yuyao Wang, Yingjie Zhang, Yidan Zhang, Jing Ji, Hao Sun, Min Luo, Ping Gu
Age-related macular degeneration (AMD) is associated with the dysfunction and death of the retinal pigment epithelium (RPE). Recently, there has been increasing interest in stem cell-derived RPE cells for cell replacement therapies, such as those for AMD. The present study investigated whether RPE-conditioned medium (RPECM) could promote the differentiation of human adipose tissue-derived mesenchymal stromal cells (hADSCs) into RPE cells, and enhance the proliferation and migration of these cells. Reverse-transcription quantitative polymerase chain reaction analysis demonstrated that RPECM induced hADSCs to differentiate into cells expressing RPE markers, including retinoid isomerohydrolase (RPE65), cytokeratin (CK8) and Bestrophin, which were identified to be significantly upregulated by ~10-fold, 3...
October 2017: Experimental and Therapeutic Medicine
https://www.readbyqxmd.com/read/29038443/phylogenetic-analysis-of-the-metazoan-carotenoid-oxygenase-superfamily-a-new-ancestral-gene-assemblage-of-bco-like-bcol-proteins
#7
Eugenia Poliakov, Joseph Soucy, Susan Gentleman, Igor B Rogozin, T Michael Redmond
Here we describe a new family of carotenoid cleavage oxygenases (CCOs) in metazoans, the BCO2-like (BCOL) clade, which contains lancelet, nematode, and molluscan carotenoid oxygenase sequences. Phylogenetic analysis of CCOs in all kingdoms of life confirmed that the BCOL enzymes are an independent clade of ancient origin. One of the predicted lancelet BCOL proteins, cloned and analyzed for carotenoid cleavage activity in a bacterial carotenoid expression system, had activity similar to lancelet BCO2 proteins, although with a preference for cis isomers...
October 16, 2017: Scientific Reports
https://www.readbyqxmd.com/read/29038159/loss-of-erk1-2-in-the-retinal-pigment-epithelium-leads-to-rpe65-decrease-and-retinal-degeneration
#8
Aswin Pyakurel, Delphine Balmer, Marc K Saba-El-Leil, Caroline Kizilyaprak, Jean Daraspe, Bruno M Humbel, Laure Voisin, Yun Z Le, Johannes von Lintig, Sylvain Meloche, Raphaël Roduit
Recent work has suggested that the activity of extracellular signal-regulated kinase (ERK) 1/2 is increased in the retinal pigment epithelium (RPE) of age-related macular degeneration (ARMD) patients and therefore could be an attractive therapeutic target. Notably, ERK1/2 pathway inhibitors are used in cancer therapy, with severe and non-characterized ocular side effects. To decipher the role of ERK1/2 in RPE cells, we conditionally disrupted the Erk1 and Erk2 genes in the mouse RPE. Loss of ERK1/2 activity resulted in a significant decrease of RPE65 expression, a decrease in ocular retinoid levels concomitant with low visual function and a rapid disorganization of RPE cells, ultimately leading to retinal degeneration...
October 16, 2017: Molecular and Cellular Biology
https://www.readbyqxmd.com/read/29033008/safety-and-long-term-efficacy-of-aav4-gene-therapy-in-patients-with-rpe65-leber-congenital-amaurosis
#9
Guylène Le Meur, Pierre Lebranchu, Fanny Billaud, Oumeya Adjali, Sébastien Schmitt, Stéphane Bézieau, Yann Péréon, Romain Valabregue, Catherine Ivan, Christophe Darmon, Philippe Moullier, Fabienne Rolling, Michel Weber
The aim of this study was the evaluation of the safety and efficacy of unilateral subretinal injection of the adeno-associated vector (AAV) serotypes 2 and 4 (AAV2/4) RPE65-RPE65 vector in patients with Leber congenital amaurosis (LCA) associated with RPE65 gene deficiency. We evaluated ocular and general tolerance and visual function up to 1 year after vector administration in the most severely affected eye in nine patients with retinal degeneration associated with mutations in the RPE65 gene. Patients received either low (1...
September 19, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28985805/antigen-specificity-of-antiretinal-antibodies-in-patients-with-noninfectious-uveitis
#10
Ebrima Gibbs, Joanne Matsubara, Sijia Cao, Jing Cui, Farzin Forooghian
OBJECTIVE: Antiretinal antibodies (ARAs) have previously been described in noninfectious uveitis. However, the antigen specificity of these ARAs has not been investigated. The purpose of this study was to identify antigen-specific ARAs in noninfectious uveitis. METHODS: A total of 18 patients with noninfectious uveitis were enrolled. Surface plasmon resonance was used to measure binding responses of patient and control sera against several uveitogenic proteins: recoverin, S-antigen, interphotoreceptor retinoid binding (IRBP), retinal-pigment-epithelium-specific 65-kDa protein (RPE65), tyrosinase-related protein 1 (TRYP1), and tyrosinase-related protein 2 (TRYP2)...
October 2017: Canadian Journal of Ophthalmology. Journal Canadien D'ophtalmologie
https://www.readbyqxmd.com/read/28973952/rpe65-takes-on-another-role-in-the-vertebrate-retina
#11
T Michael Redmond
No abstract text is available yet for this article.
October 10, 2017: Proceedings of the National Academy of Sciences of the United States of America
https://www.readbyqxmd.com/read/28962184/bioinformatic-analysis-of-retinal-gene-function-and-expression-in-diabetic-rats
#12
Wenjuan Zhao, Dong Wang, Jun Zhao, Wenqing Zhao
The aim of the present study was to investigate the changes in retinal gene expression at three time points and assess the underlying molecular mechanisms of diabetic retinopathy (DR) in a streptozotocin (STZ)-induced diabetes rat model using bioinformatics analysis. The gene expression profile of GSE28831 was extracted from the Gene Expression Omnibus database and differentially expressed genes (DEGs) were identified at three different time points (1, 4 and 12 weeks) using the limma package in R language. Gene ontology (GO) enrichment analysis of DEGs was performed followed by a principal component and pathway enrichment analysis of the selected DEGs along with protein-protein interaction network construction at the three time points...
September 2017: Experimental and Therapeutic Medicine
https://www.readbyqxmd.com/read/28955745/bioengineered-bruch-s-like-extracellular-matrix-promotes-retinal-pigment-epithelial-differentiation
#13
Samuel McLenachan, Erwei Hao, Dan Zhang, Ling Zhang, Michael Edel, Fred Chen
In the eye, the retinal pigment epithelium (RPE) adheres to a complex protein matrix known as Bruch's membrane (BrM). The aim of this study was to provide enriched conditions for RPE cell culture through the production of a BrM-like matrix. Our hypothesis was that a human RPE cell line would deposit an extracellular matrix (ECM) resembling BrM. The composition and structure of ECM deposited by ARPE19 cells (ARPE19-ECM) was characterized. To produce ARPE19-ECM, ARPE19 cells were cultured in the presence dextran sulphate...
July 2017: Biochemistry and Biophysics Reports
https://www.readbyqxmd.com/read/28945494/identification-of-the-genetic-determinants-responsible-for-retinal-degeneration-in-families-of-mexican-descent
#14
Adda Villanueva, Pooja Biswas, Kameron Kishaba, John Suk, Keerti Tadimeti, Pongali B Raghavendra, Karine Nadeau, Bruno Lamontagne, Lambert Busque, Steve Geoffroy, Ian Mongrain, Géraldine Asselin, Sylvie Provost, Marie-Pierre Dubé, Eric Nudleman, Radha Ayyagari
PURPOSE: To investigate the clinical characteristics and genetic basis of inherited retinal degeneration (IRD) in six unrelated pedigrees from Mexico. METHODS: A complete ophthalmic evaluation including measurement of visual acuities, Goldman kinetic or Humphrey dynamic perimetry, Amsler test, fundus photography, and color vision testing was performed. Family history and blood samples were collected from available family members. DNA from members of two pedigrees was examined for known mutations using the APEX ARRP genotyping microarray and one pedigree using the APEX LCA genotyping microarray...
September 25, 2017: Ophthalmic Genetics
https://www.readbyqxmd.com/read/28943836/evaluating-efficiencies-of-dual-aav-approaches-for-retinal-targeting
#15
Livia S Carvalho, Heikki T Turunen, Sarah J Wassmer, María V Luna-Velez, Ru Xiao, Jean Bennett, Luk H Vandenberghe
Retinal gene therapy has come a long way in the last few decades and the development and improvement of new gene delivery technologies has been exponential. The recent promising results from the first clinical trials for inherited retinal degeneration due to mutations in RPE65 have provided a major breakthrough in the field and have helped cement the use of recombinant adeno-associated viruses (AAV) as the major tool for retinal gene supplementation. One of the key problems of AAV however, is its limited capacity for packaging genomic information to a maximum of around 4...
2017: Frontiers in Neuroscience
https://www.readbyqxmd.com/read/28889993/a-subpopulation-of-activated-retinal-macrophages-selectively-migrated-to-regions-of-cone-photoreceptor-stress-but-had-limited-effect-on-cone-death-in-a-mouse-model-for-type-2-leber-congenital-amaurosis
#16
Peter H Tang, Mark J Pierson, Neal D Heuss, Dale S Gregerson
BACKGROUND: Studies of antigen presentation in retina using mice that expressed green fluorescent protein (GFP) from a transgenic CD11c promoter found that retinal GFP(hi) cells possessed antigen presentation function. Subsequent studies found that these high GFP(hi) cells preferentially localized to sites of retinal injury, consistent with their APC function. Interest in the roles of macrophages in degenerative CNS diseases led us to study the GFP(hi) cells in a retinal model of neurodegeneration...
December 2017: Molecular and Cellular Neurosciences
https://www.readbyqxmd.com/read/28880021/long-term-expression-of-melanopsin-and-channelrhodopsin-causes-no-gross-alterations-in-the-dystrophic-dog-retina
#17
B Ameline, K-T Tshilenge, M Weber, M Biget, L Libeau, R Caplette, A Mendes-Madeira, N Provost, C Guihal, S Picaud, P Moullier, V Pichard, T Cronin, C Isiegas
Several preclinical studies have investigated the potential of algal channelrhodopsin and human melanopsin as optogenetic tools for vision restoration. In the present study, we assessed the potentially deleterious effects of long-term expression of these optogenes on the diseased retina in a large animal model of retinal degeneration, the RPE65-deficient Briard dog model of Leber congenital amaurosis. Intravitreal injection of adeno-associated virus vectors expressing channelrhodopsin and melanopsin had no effect on retinal thickness over a 16-month period post injection...
September 7, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28874556/rpe65-has-an-additional-function-as-the-lutein-to-meso-zeaxanthin-isomerase-in-the-vertebrate-eye
#18
Rajalekshmy Shyam, Aruna Gorusupudi, Kelly Nelson, Martin P Horvath, Paul S Bernstein
Carotenoids are plant-derived pigment molecules that vertebrates cannot synthesize de novo that protect the fovea of the primate retina from oxidative stress and light damage. meso-Zeaxanthin is an ocular-specific carotenoid for which there are no common dietary sources. It is one of the three major carotenoids present at the foveal center, but the mechanism by which it is produced in the eye is unknown. An isomerase enzyme is thought to be responsible for the transformation of lutein to meso-zeaxanthin by a double-bond shift mechanism, but its identity has been elusive...
October 10, 2017: Proceedings of the National Academy of Sciences of the United States of America
https://www.readbyqxmd.com/read/28754419/evaluation-of-tolerance-to-lentiviral-lv-rpe65-gene-therapy-vector-after-subretinal-delivery-in-non-human-primates
#19
Alexandre Matet, Corinne Kostic, Alexis-Pierre Bemelmans, Alexandre Moulin, Serge G Rosolen, Samia Martin, Fulvio Mavilio, Vazrik Amirjanians, Knut Stieger, Birgit Lorenz, Francine Behar-Cohen, Yvan Arsenijevic
Several approaches have been developed for gene therapy in RPE65-related Leber congenital amaurosis. To date, strategies that have reached the clinical stages rely on adeno-associated viral vectors and two of them documented limited long-term effect. We have developed a lentiviral-based strategy of RPE65 gene transfer that efficiently restored protein expression and cone function in RPE65-deficient mice. In this study, we evaluated the ocular and systemic tolerances of this lentiviral-based therapy (LV-RPE65) on healthy nonhuman primates (NHPs), without adjuvant systemic anti-inflammatory prophylaxis...
October 2017: Translational Research: the Journal of Laboratory and Clinical Medicine
https://www.readbyqxmd.com/read/28712537/efficacy-and-safety-of-voretigene-neparvovec-aav2-hrpe65v2-in-patients-with-rpe65-mediated-inherited-retinal-dystrophy-a-randomised-controlled-open-label-phase-3-trial
#20
Stephen Russell, Jean Bennett, Jennifer A Wellman, Daniel C Chung, Zi-Fan Yu, Amy Tillman, Janet Wittes, Julie Pappas, Okan Elci, Sarah McCague, Dominique Cross, Kathleen A Marshall, Jean Walshire, Taylor L Kehoe, Hannah Reichert, Maria Davis, Leslie Raffini, Lindsey A George, F Parker Hudson, Laura Dingfield, Xiaosong Zhu, Julia A Haller, Elliott H Sohn, Vinit B Mahajan, Wanda Pfeifer, Michelle Weckmann, Chris Johnson, Dina Gewaily, Arlene Drack, Edwin Stone, Katie Wachtel, Francesca Simonelli, Bart P Leroy, J Fraser Wright, Katherine A High, Albert M Maguire
BACKGROUND: Phase 1 studies have shown potential benefit of gene replacement in RPE65-mediated inherited retinal dystrophy. This phase 3 study assessed the efficacy and safety of voretigene neparvovec in participants whose inherited retinal dystrophy would otherwise progress to complete blindness. METHODS: In this open-label, randomised, controlled phase 3 trial done at two sites in the USA, individuals aged 3 years or older with, in each eye, best corrected visual acuity of 20/60 or worse, or visual field less than 20 degrees in any meridian, or both, with confirmed genetic diagnosis of biallelic RPE65 mutations, sufficient viable retina, and ability to perform standardised multi-luminance mobility testing (MLMT) within the luminance range evaluated, were eligible...
August 26, 2017: Lancet
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