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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High throughput screening for discovery of materials that control stem cell fate
Asha K. Patel,Asha K. Patel,Mark W. Tibbitt,Adam D. Celiz,Adam D. Celiz,Martyn C. Davies,Robert Langer,Chris Denning,Morgan R. Alexander,Daniel G. Anderson +9 more
TL;DR: In this article, the authors present screening platforms for the discovery of material properties that influence stem cell behavior, such as chemistry, topography, and stiffness, in two and three-dimensional environments.
Journal ArticleDOI
Progressing a human embryonic stem-cell-based regenerative medicine therapy towards the clinic
TL;DR: The current status of the development of embryonic stem-cell-based regenerative medicine therapies with a focus upon retinal disease is discussed, and some of the own experiences of progressing a retinal pigment epithelium cell replacement therapy towards the clinic are highlighted.
Journal ArticleDOI
The genetics of age-related macular degeneration (AMD)--Novel targets for designing treatment options?
TL;DR: It is pointed out that known genetic factors associated with AMD govern the risk to develop disease and thus may not play a role in its severity or progression and treatments based on such knowledge appear appropriate rather for prevention than treatment of manifest disease.
Journal ArticleDOI
Treatment Paradigms for Retinal and Macular Diseases Using 3-D Retina Cultures Derived From Human Reporter Pluripotent Stem Cell Lines.
Rossukon Kaewkhaw,Rossukon Kaewkhaw,Manju Swaroop,Kohei Homma,Jutaro Nakamura,Matthew Brooks,Koray Dogan Kaya,Vijender Chaitankar,Sam Michael,Gregory J. Tawa,Jizhong Zou,Mahendra Rao,Wei Zheng,Tiziana Cogliati,Anand Swaroop +14 more
TL;DR: The use of pluripotent stem cell lines carrying fluorescent reporters driven by retinal promoters to derive three-dimensional retina in culture is discussed and how this system can be exploited for elucidating human retinal biology, creating disease models in a dish, and designing targeted drug screens for retinal and macular degeneration.
Journal ArticleDOI
Intravitreal Administration of Human Bone Marrow CD34+ Stem Cells in a Murine Model of Retinal Degeneration.
Elad Moisseiev,Zeljka Smit-McBride,Sharon L. Oltjen,Pengfei Zhang,Robert J. Zawadzki,Monica J. Motta,Christopher J. Murphy,Whitney Cary,Geralyn Annett,Jan A. Nolta,Susanna S Park +10 more
TL;DR: Although a functional benefit of this cell therapy was not seen on ERG in this rapidly progressive retinal degeneration model, molecular changes in the retina associated with CD34+ cell therapy suggest potential trophic regenerative effects that warrant further exploration.
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.
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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