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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Insights from Genetic Model Systems of Retinal Degeneration: Role of Epsins in Retinal Angiogenesis and VEGFR2 Signaling.
TL;DR: Several well-characterized and commonly used animal models are reviewed and their contributions to the understanding of the mechanisms of retinal degeneration and thereby providing novel treatment options including gene therapy, stem cell therapy, nanomedicine, and CRISPR/Cas9 genome editing are reviewed.
Journal ArticleDOI
Cell therapy for macular degeneration—first phase I/II pluripotent stem cell-based clinical trial shows promise
ZhiGuo Chen,Y. Alex Zhang +1 more
TL;DR: The long awaited medium-to long-term results of the trials that came out in a recent Lancet publication confirmed that hESC-derived cells improved vision in macular degenera-tion patients while showing no apparent safety concerns, bringing much relief to some and considerable excitement to many in the stem cell field.
Journal ArticleDOI
Pluripotent Stem Cell-Based Organoid Technologies for Developing Next-Generation Vision Restoration Therapies of Blindness.
Ratnesh K. Singh,Francois Binette,Magdalene J. Seiler,Simon M. Petersen-Jones,Igor O. Nasonkin +4 more
TL;DR: This review will outline major regenerative medicine approaches, which are emerging as promising for preserving or/and restoring vision and discuss the potential of each of these approaches to reach commercialization step and be converted to treatments, which could at least ameliorate blindness caused by retinal cell death.
Journal ArticleDOI
Patch grafting, strategies for transplantation of organoids into solid organs such as liver.
Zhang Wencheng,Zhang Wencheng,Giacomo Lanzoni,Homayoun Hani,Diletta Overi,Vincenzo Cardinale,Sean G Simpson,Wendy A. Pitman,Amanda Allen,Xianwen Yi,Xicheng Wang,David A. Gerber,Glenn D. Prestwich,Oswaldo A. Lozoya,Eugenio Gaudio,Domenico Alvaro,Debra Tokaz,Juan Domínguez-Bendala,Christopher A. Adin,Jorge A. Piedrahita,Kyle G. Mathews,Praveen Sethupathy,Guido Carpino,Zhiying He,Eliane Wauthier,Lola M. Reid +25 more
TL;DR: In this article, the porcine liver-derived mesenchymal stem cells (MSCs) were co-transplanted with donor mature cells, and the results showed that the transplanted organ's clearance of hyaluronans enabled restoration of tissue-specific paracrine and systemic signaling, resulting in normal hepatic histology.
Journal ArticleDOI
Adult Human Peripheral Blood Mononuclear Cells Are Capable of Producing Neurocyte or Photoreceptor-Like Cells That Survive in Mouse Eyes After Preinduction With Neonatal Retina.
Bikun Xian,Yichi Zhang,Yuting Peng,Jianfa Huang,Weihua Li,Wencong Wang,Min Zhang,Kaijing Li,Hening Zhang,Minglei Zhao,Xing Liu,Bing Huang +11 more
TL;DR: The ease of harvesting, viability in vivo, capacity to express neuronal and photoreceptor proteins, and capacity for functional enhancement suggest that hPBMCs are potential candidates for cell replacement therapy to treat retinal degenerative diseases.
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.
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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.
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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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