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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Book ChapterDOI
Developing Cell-Based Therapies for RPE-Associated Degenerative Eye Diseases.
TL;DR: This chapter will focus on the different cell-based strategies developed in the past and current approaches to prevent photoreceptor death in RPE-associated degenerative eye diseases.
Journal ArticleDOI
Beneath the sword of Damocles: regenerative medicine and the shadow of immunogenicity
Paul J. Fairchild,Christopher Horton,Priyoshi Lahiri,Kumaran Shanmugarajah,Timothy J. Davies +4 more
TL;DR: The evidence on which contradictory claims about the immunogenicity of cell types and tissues differentiated from pluripotent stem cells are based is reviewed to reach an objective assessment of the likely magnitude of the immunological challenges ahead.
Journal ArticleDOI
Current status of stem cell therapy: opportunities and limitations
Elena Rusu,Laura Necula,Ana Iulia Neagu,Mihail Alecu,Cristian Stan,Radu Albulescu,Cristiana Tanase +6 more
TL;DR: Although the preliminary results are interesting, further studies are required in order to evaluate the safety and benefits of stem cell therapy, considering the teratoma development and ethical considerations in embryonic stem cell cases or reprogramming-induced somatic mutations and epigenetic defects.
Book ChapterDOI
Pluripotent Stem Cells to Model Degenerative Retinal Diseases: The RPE Perspective
TL;DR: The utility of patient-derived hiPSCs for establishment of human cell models and molecular and pharmacological studies on patient- derived cell models of retinal degenerative diseases where RPE cellular defects play a major pathogenic role in disease development and progression are discussed.
Journal ArticleDOI
Access to stem cell data and registration of pluripotent cell lines: The Human Pluripotent Stem Cell Registry (hPSCreg).
Nancy Mah,Stefanie Seltmann,B. Aran,Rachel Steeg,Johannes Dewender,Nils Bultjer,Anna Veiga,Glyn Stacey,Andreas Kurtz +8 more
TL;DR: A short primer on the history of stem cell-based products is presented, the ethical and regulatory issues introduced in the course of working with hPSC-derived products and their associated data are summarized, and the Human Pluripotent Stem Cell Registry is presented as a valuable resource for all stakeholders in therapies and disease modeling based on hPSCs.
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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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