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
Advantages of the Eye as a Target Organ for Cell-Based Therapy in the Central Nervous System
TL;DR: The eye is an immune privileged organ, particularly the subretinal space, which may reduce the need for long-term immune suppression of transplant recipients, and high-resolution noninvasive imaging technology permits visualization and functional assessment of the transplanted cells in situ.
Journal ArticleDOI
Global Transcriptional and Epigenetic Reconfiguration during Chemical Reprogramming of Human Retinal Pigment Epithelial Cells into Photoreceptor-like Cells
TL;DR: RPE cells can be efficiently reprogrammed into photoreceptor-like cells through defined pharmacological modulations, providing a useful cell source forPhotoreceptor generation in cell replacement therapy for retinal degenerative diseases.
Book ChapterDOI
Surgical Approaches for Cell Transplantation in Cell Replacement Therapy
TL;DR: Important surgical approaches to hESC-RPE transplantation for age-related macular degeneration and Stargardt’s disease are summarized and the future directions of cell therapy in degenerative retinal disease are discussed.
Journal ArticleDOI
Stem cells pass safety test in vision-loss trial
TL;DR: Treatment developed by company with tumultuous history is set for larger studies, paving the way for further studies in animals and humans.
Book ChapterDOI
Stem Cell-Derived Retinal Cells for Transplantation
TL;DR: Stem cell-based therapies that introduce healthy tissue into the eye have been shown to restore or rescue the degenerated retina and improve vision in animal models as discussed by the authors, which is a promising treatment for retinal diseases.
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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Michiko Mandai,Akira Watanabe,Yasuo Kurimoto,Yasuhiko Hirami,Chikako Morinaga,Takashi Daimon,Masashi Fujihara,Hiroshi Akimaru,Noriko Sakai,Yumiko Shibata,Motoki Terada,Yui Nomiya,Shigeki Tanishima,Masahiro Nakamura,Hiroyuki Kamao,Sunao Sugita,Akishi Onishi,Tomoko Ito,Kanako Fujita,Shin Kawamata,Masahiro J. Go,Chikara Shinohara,Kenichiro Hata,Masanori Sawada,Midori Yamamoto,Sachiko Ohta,Yasuo Ohara,Kenichi Yoshida,Junko Kuwahara,Yuko Kitano,Naoki Amano,Masafumi Umekage,Fumiyo Kitaoka,Azusa Tanaka,Chihiro Okada,Naoko Takasu,Seishi Ogawa,Shinya Yamanaka,Masayo Takahashi +38 more