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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Journal ArticleDOI
Stargardt disease – what general ophthalmologists should know about this macular dystrophy
TL;DR: Stargardt disease is the most common autosomal recessive childhood and adulthood macular degeneration, caused by mutations in the ABCA4 gene located on chromosome 1 and the consequence of mutation is the accumulation of a lipofuscin component known as A2E, toxic for retinal pigment epithelium and photoreceptors.
Book ChapterDOI
Stem Cell Biology and Its Clinical Application
TL;DR: Stem cells are cells which regardless of their source have the properties of division and renewing for long periods through mitotic cell division, are dedifferentiated (unspecialized) with unique capacity to produce unaltered daughter cells (self-renewal), and generate specialized cell types under certain physiologic or experimental conditions.
Journal ArticleDOI
Sourcing photoreceptor-like cells for treating vision loss
TL;DR: A new, albeit tentative, strategy to rectify the loss of photoreceptor cells is presented, which is a critical event in loss of vision.
Journal ArticleDOI
Les cellules souches pluripotentes dans le traitement de l’insuffisance cardiaque - Statut actuel, problèmes et perspectives
Manon Desgres,Philippe Menasché +1 more
TL;DR: In this article, a revue discute l'etat actuel de la recherche dans ce domaine, les problemes qui restent a resoudre and les approches susceptibles d’accelerer les applications cliniques of ce type cellulaire.
Journal ArticleDOI
Stem cell-derived retinal pigment epithelium cell therapy: Past and future directions
Landon J. Rohowetz,Peter Koulen +1 more
TL;DR: In this article , a review of stem-cell derived retinal pigment epithelium (RPE) cell transplantation for age-related macular degeneration (AMD) is presented.
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.
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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