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Akademický článek

Restoration of visual function in advanced disease after transplantation of purified human pluripotent stem cell-derived cone photoreceptors

Publikováno v: Cell Reports, Vol 35, Iss 3, Pp 109022- (2021)

Summary: Age-related macular degeneration and other macular diseases result in the loss of light-sensing cone photoreceptors, causing irreversible sight impairment. Photoreceptor replacement may restore vision by transplanting healthy cells, which mu

Externí odkaz: https://doaj.org/article/f2a1aa9c1fb544b5b5376bb03b90e605

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RNAi‐mediated suppression of vimentin or glial fibrillary acidic protein prevents the establishment of Müller glial cell hypertrophy in progressive retinal degeneration

Autor: Robin R. Ali, Anna B. Graca, James W B Bainbridge, Alexander J. Smith, Ayako Matsuyama, Joana Ribeiro, Rachael A. Pearson, Mark Basche, Nozie D. Aghaizu, Aikaterini A Kalargyrou, Anastasios Georgiadis, Claire Hippert

Publikováno v: Glia. 69:2272-2290

Gliosis is a complex process comprising upregulation of intermediate filament (IF) proteins, particularly glial fibrillary acidic protein (GFAP) and vimentin, changes in glial cell morphology (hypertrophy) and increased deposition of inhibitory extra

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6d345deb36e380d06c43502d6b5e77d5
https://doi.org/10.1002/glia.24034

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Nanotube‐like processes facilitate material transfer between photoreceptors

Autor: Aikaterini A Kalargyrou, Emma L. West, Alexander J. Smith, Mark Basche, Robin R. Ali, Rachael A. Pearson, Aura Hare

Publikováno v: EMBO reports. 22

Neuronal communication is typically mediated via synapses and gap junctions. New forms of intercellular communication, including nanotubes (NTs) and extracellular vesicles (EVs), have been described for non-neuronal cells, but their role in neuronal

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::2cc3ad0a73357b1a3fc1b6ead8cffc13
https://doi.org/10.15252/embr.202153732

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Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease

Autor: Robin R. Ali, Amna Z. Shah, Giulia Massaro, Sara E. Mole, Olha Semenyuk, Ryea Maswood, Michael P. Hughes, Sophia-Martha Kleine Holthaus, Alexander J. Smith, Mark Basche, Saul Herranz-Martin, Justin Hoke, Ahad A. Rahim, Mikel Aristorena, Izabela P Klaska

Publikováno v: Human molecular genetics. 28(23)

The neuronal ceroid lipofuscinoses (NCLs), more commonly referred to as Batten disease, are a group of inherited lysosomal storage disorders that present with neurodegeneration, loss of vision and premature death. There are at least 13 genetically di

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d6ee98cc75a5fe0d91ad95cb7e646534
https://pubmed.ncbi.nlm.nih.gov/31807779

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Restoration of visual function in advanced disease after transplantation of purified human pluripotent stem cell-derived cone photoreceptors

Publikováno v: Cell Reports
Cell Reports, Vol 35, Iss 3, Pp 109022-(2021)

Summary Age-related macular degeneration and other macular diseases result in the loss of light-sensing cone photoreceptors, causing irreversible sight impairment. Photoreceptor replacement may restore vision by transplanting healthy cells, which mus

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9d7ff1187122a7b66381120016057aff
https://doi.org/10.1016/j.celrep.2021.109022

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Sustained and Widespread Gene Delivery to the Corneal Epithelium via In Situ Transduction of Limbal Epithelial Stem Cells, Using Lentiviral and Adeno-Associated Viral Vectors

Autor: D. Frank P. Larkin, Alexander J. Smith, Mark Basche, Satoshi Kawasaki, Matthew J. Branch, Daniel Kampik, Robin R. Ali, Martha Robinson

Publikováno v: Human gene therapy. 29(10)

Corneal epithelial dystrophies are typically characterized by symptoms such as pain, light sensitivity, and corneal opacification leading to impaired vision. The development of gene therapy for such conditions has been hindered by an inability to ach

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a5b1894df234fb3ab4073ad9bd5e7e06
https://pubmed.ncbi.nlm.nih.gov/30070149

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A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency

Publikováno v: Brain
Tordo, J, O'Leary, C, Antunes, A S L M, Palomar, N, Aldrin-Kirk, P, Basche, M, Bennett, A, D'Souza, Z, Gleitz, H, Godwin, A, Holley, R J, Parker, H, Liao, A Y, Rouse, P, Youshani, A S, Dridi, L, Martins, C, Levade, T, Stacey, K B, Davis, D M, Dyer, A, Clément, N, Björklund, T, Ali, R R, Agbandje-McKenna, M, Rahim, A A, Pshezhetsky, A, Waddington, S N, Linden, R M, Bigger, B W & Henckaerts, E 2018, ' A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency ', Brain, vol. 141, no. 7, pp. 2014-2031 . https://doi.org/10.1093/brain/awy126
Tordo, J, O'Leary, C, Antunes, A S L M, Palomar, N, Aldrin-Kirk, P, Basche, M, Bennett, A, D'Souza, Z, Gleitz, H, Godwin, A, Holley, R, Parker, H, Liao, A, Rouse, P, Youshani, A, Dridi, L, Martins, C, Levade, T, Stacey, K, Davis, D M, Dyer, A, Clement, N, Bjorklund, T, Ali, R R, Agbandje-McKenna, M, Rahim, A, Pshezhetsky, A V, Waddington, S N, Linden, R M, Bigger, B & Henckaerts, E 2018, ' A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency ', Brain, vol. 141, no. 7, pp. 2014-2031 . https://doi.org/10.1093/brain/awy126

Tordo et al. present a novel AAV gene therapy vector, AAV-TT, which exceeds the current benchmark neurotropic serotypes AAV9 and AAVrh10 and enables unprecedented correction of a lysosomal transmembrane enzyme deficiency. AAV-TT based gene therapies

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5b17a7608215d5ccd1f1e0191c5e9330

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Modulation of Contact Inhibition by ZO-1/ZONAB Gene Transfer—A New Strategy to Increase the Endothelial Cell Density of Corneal Grafts

Autor: Robin R. Ali, D. Frank P. Larkin, Mark Basche, Ulrich F O Luhmann, Alexander J. Smith, Anastasios Georgiadis, Daniel Kampik

Publikováno v: Investigative Opthalmology & Visual Science. 60:3170

Purpose Endothelial cell density (ECD) is the principal factor determining the success of corneal transplants. Here we explored a strategy to increase corneal ECD in human explants via modulation of the ZO-1/ZONAB pathway. In multiple cell types, ZO-

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::12f6b8d3ccba4df2331451fc30305295
https://doi.org/10.1167/iovs.18-26260

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Successful Gene Therapy in Older Rpe65-Deficient Dogs Following Subretinal Injection of an Adeno-Associated Vector Expressing RPE65

Autor: Joshua T. Bartoe, Freya M. Mowat, Matthew J. Annear, Selina A. Azam, Robin R. Ali, Alexander J. Smith, Paul G. Curran, Simon M. Petersen-Jones, Janice Querubin, James W B Bainbridge, Mark Basche

Publikováno v: Human Gene Therapy. 24:883-893

Young Rpe65-deficient dogs have been used as a model for human RPE65 Leber congenital amaurosis (RPE65-LCA) in proof-of-concept trials of recombinant adeno-associated virus (rAAV) gene therapy. However, there are relatively few reports of the outcome

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7530ada961dde910f20256eb1f2de3d6
https://doi.org/10.1089/hum.2013.146

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