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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PSCs Reveal PUFA-Provoked Mitochondrial Stress as a Central Node Potentiating RPE Degeneration in Bietti's Crystalline Dystrophy.
Zhao Zhang,Zhao Zhang,Bin Yan,Bin Yan,Fei Gao,Qing Li,Xiaohong Meng,Peikai Chen,Lei Zhou,Wen Deng,Cheng Li,Weiyi Xu,Shuo Han,Hong Feng,Yaping Li,J. Chen,Zhengqin Yin,Can Liao,Hung-Fat Tse,Aimin Xu,Qizhou Lian,Qizhou Lian +21 more
TL;DR: In this article, the PUFA overload increased reactive oxygen species, impaired mitochondrial respiratory functions, and triggered mitochondrial stress-activated p53-independent apoptosis in CYP4V2 mutant RPE cells.
Journal ArticleDOI
Leber congenital amaurosis/early-onset severe retinal dystrophy: current management and clinical trials.
Malena Daich Varela,Malena Daich Varela,Thales Antonio Cabral de Guimaraes,Thales Antonio Cabral de Guimaraes,Michalis Georgiou,Michalis Georgiou,Michel Michaelides,Michel Michaelides +7 more
TL;DR: A review of the current management of Leber congenital amaurosis (LCA) can be found in this paper, where the authors highlight the clinical trials that are on-going and planned.
Journal ArticleDOI
Transplantation of rat embryonic stem cell-derived retinal progenitor cells preserves the retinal structure and function in rat retinal degeneration
Zepeng Qu,Yuan Guan,Lu Cui,Jian Song,Junjie Gu,Hanzhi Zhao,Lei Xu,Lixia Lu,Ying Jin,Ying Jin,Guo-Tong Xu +10 more
TL;DR: The retinal neuron enriched rESC-RPC2 protected the structure and function of retina in rats with genetic retinal degeneration and could be a candidate cell source for treating some degenerative retinal diseases in human trials.
Journal ArticleDOI
Stem Cell Therapy for Treatment of Ocular Disorders
Padma Priya Sivan,Sakinah Syed,Pooi Ling Mok,Akon Higuchi,Kadarkarai Murugan,Abdullah A. Alarfaj,Murugan A. Munusamy,Rukman Awang Hamat,Akihiro Umezawa,S. Suresh Kumar +9 more
TL;DR: The processes of human eye development are reviewed and the strategies that have been exploited in an effort to regain visual function in the preclinical and clinical state are addressed.
Journal ArticleDOI
Retinal degeneration rat model: A study on the structural and functional changes in the retina following injection of sodium iodate.
Avin Ee-Hwan Koh,Hiba Amer Alsaeedi,Munirah Binti Abd Rashid,Chenshen Lam,Mohd Hairul Nizam Harun,Muhamad Fakhri bin Mohd Saleh,Chi D Luu,S. Suresh Kumar,Min Hwei Ng,Hazlita Mohd Isa,Sue Ngein Leow,Kong Yong Then,Mae-Lynn Catherine Bastion,Mohammed Safwan Ali Khan,Pooi Ling Mok +14 more
TL;DR: The pathological findings in this model reveal a degenerating retina, and can be further utilized to develop effective therapies for RPE, photoreceptor, and bipolar cell regeneration.
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.
Journal ArticleDOI
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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