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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Adult human RPE for transplantation: renewing an old promise
TL;DR: New advances in using adult RPE, which have renewed their promise in treating RPE-related eye diseases are summarized.
Journal ArticleDOI
Pluripotent stem cells: A therapeutic source for age-related macular degeneration.
TL;DR: The opportunities in the various types of cell-based therapy, their limitations, and what is possible for India are discussed.
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Transplantation of rat embryonic stem cell-derived retinal cells restores visual function in the Royal College of Surgeons rats
TL;DR: The developed stepwise protocol can derive retinal cells from ESCs and can restore visual function of RCS rats, providing evidence for potential clinical application of ESC-based cell therapy for retinal degeneration.
Journal ArticleDOI
Therapeutic Options Under Development for Nonneovascular Age-Related Macular Degeneration and Geographic Atrophy.
Joon-Bom Kim,Eleonora M. Lad +1 more
TL;DR: In this paper, a review of the current management and treatment of non-neovascular age-related macular degeneration, the drugs and devices that have been under investigation for the treatment of geographic atrophy, and the latest clinical trial results is presented.
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Reprogramming of Differentiated Mammalian and Human Retinal Pigment Epithelium: Current Achievements and Prospects
TL;DR: This review focuses on research in gene-directed reprogramming of vertebrate RPE cells into retinal neurons, with detailed analysis of the genes used as the main reprograming factors, comparative analysis, and extrapolation of experimental data from animals to humans.
References
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Journal ArticleDOI
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.
Journal ArticleDOI
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.
Journal ArticleDOI
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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