Journal ArticleDOI
Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies
Steven D. Schwartz,Carl D. Regillo,Byron L. Lam,Dean Eliott,Philip J. Rosenfeld,Ninel Z. Gregori,Jean-Pierre Hubschman,Janet L. Davis,Gad Heilwell,Marc J. Spirn,Joseph I. Maguire,Jane Bateman,Rosaleen Ostrick,Debra Morris,Matthew Vincent,Eddy Anglade,Lucian V. Del Priore,Robert Lanza +17 more
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TLDR
The results suggest that hESC-derived cells could provide a potentially safe new source of cells for the treatment of various unmet medical disorders requiring tissue repair or replacement.About:
This article is published in The Lancet.The article was published on 2015-02-07. It has received 990 citations till now. The article focuses on the topics: Macular dystrophy & Stargardt disease.read more
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Epiretinal membrane formation after intravitreal autologous stem cell implantation in a retinitis pigmentosa patient.
TL;DR: This is the first report of ERM formation following intravitreal autologous stem cells injection in a retinitis pigmentosa patient and CD34-positive stem cells were detected in a human eye at 4 months after injection.
Journal ArticleDOI
Mislocalisation of BEST1 in iPSC-derived retinal pigment epithelial cells from a family with autosomal dominant vitreoretinochoroidopathy (ADVIRC).
David A. Carter,Matthew Smart,William V. G. Letton,Conor M. Ramsden,Conor M. Ramsden,Britta Nommiste,Li Li Chen,Kate Fynes,Manickam N. Muthiah,Pollyanna Goh,Amelia Lane,Michael B. Powner,Andrew R. Webster,Andrew R. Webster,Lyndon da Cruz,Lyndon da Cruz,Anthony T. Moore,Anthony T. Moore,Anthony T. Moore,Peter J. Coffey,Amanda-Jayne F. Carr +20 more
TL;DR: It is shown that BEST1 is expressed more abundantly in peripheral RPE compared to central RPE and is also expressed in cells of the developing retina during human eye development, suggesting that higher levels of mislocalised BEST1 expression in the periphery could provide a mechanism that leads to the distinct clinical phenotype observed in ADVIRC patients.
Journal ArticleDOI
Assessment of Safety and Functional Efficacy of Stem Cell-Based Therapeutic Approaches Using Retinal Degenerative Animal Models.
Tai-Chi Lin,Magdalene J. Seiler,Danhong Zhu,Paulo Falabella,David R. Hinton,Dennis O. Clegg,Mark S. Humayun,Biju B. Thomas +7 more
TL;DR: The current status of preclinical safety and efficacy studies for retinal cell replacement therapies conducted in animal models will be discussed.
Journal ArticleDOI
Concise Review: One Stone for Multiple Birds: Generating Universally Compatible Human Embryonic Stem Cells.
TL;DR: New developments in this line of research are introduced and strategies to reduce the tumorigenic concerns regarding hESCs, especially after they acquire the capability to escape immune surveillance are discussed.
Journal ArticleDOI
Clinical-scale purification of pluripotent stem cell derivatives for cell-based therapies.
Goncalo Rodrigues,Carlos A V Rodrigues,Tiago G. Fernandes,Maria Margarida Diogo,Joaquim M. S. Cabral +4 more
TL;DR: The working principles behind separation methods that have facilitated the safe and controlled application of hPSC‐derived cells in laboratory settings and pre‐clinical research are reviewed.
References
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Induction of Pluripotent Stem Cells from Adult Human Fibroblasts by Defined Factors
Kazutoshi Takahashi,Koji Tanabe,Mari Ohnuki,Megumi Narita,Tomoko Ichisaka,Kiichiro Tomoda,Shinya Yamanaka +6 more
TL;DR: It is demonstrated that iPS cells can be generated from adult human fibroblasts with the same four factors: Oct3/4, Sox2, Klf4, and c-Myc.
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Embryonic Stem Cell Lines Derived from Human Blastocysts
James A. Thomson,Joseph Itskovitz-Eldor,Sander S. Shapiro,Michelle A. Waknitz,Swiergiel Jennifer J,Vivienne S. Marshall,Jeffrey M. Jones +6 more
TL;DR: Human blastocyst-derived, pluripotent cell lines are described that have normal karyotypes, express high levels of telomerase activity, and express cell surface markers that characterize primate embryonic stem cells but do not characterize other early lineages.
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Establishment in culture of pluripotential cells from mouse embryos
TL;DR: The establishment in tissue culture of pluripotent cell lines which have been isolated directly from in vitro cultures of mouse blastocysts are reported, able to differentiate either in vitro or after innoculation into a mouse as a tumour in vivo.
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The Retinal Pigment Epithelium in Visual Function
TL;DR: This review summarizes the current knowledge of RPE functions and describes how failure of these functions causes loss of visual function.
Journal ArticleDOI
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Albert M. Maguire,Francesca Simonelli,Eric A. Pierce,Edward N. Pugh,Federico Mingozzi,Jeannette L. Bennicelli,Sandro Banfi,Kathleen A. Marshall,Francesco Testa,Enrico Maria Surace,Settimio Rossi,Arkady Lyubarsky,Valder R. Arruda,Barbara A. Konkle,Edwin M. Stone,Edwin M. Stone,Junwei Sun,Jonathan B. Jacobs,L. F. Dell'Osso,Richard W. Hertle,Jian Xing Ma,T. Michael Redmond,Xiaosong Zhu,Bernd Hauck,Olga Zelenaia,Kenneth S. Shindler,Maureen G. Maguire,J. Fraser Wright,Nicholas J. Volpe,Jennifer Wellman McDonnell,Alberto Auricchio,Katherine A. High,Katherine A. High,Jean Bennett +33 more
TL;DR: This study investigated the safety of subretinal delivery of a recombinant adeno-associated virus (AAV) carrying RPE65 complementary DNA (cDNA) and found three patients with LCA2 had an acceptable local and systemic adverse-event profile after delivery of AAV2.hRPE65v2.
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