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
In situ regeneration of retinal pigment epithelium by gene transfer of E2F2: a potential strategy for treatment of macular degenerations.
Daniel Kampik,Mark Basche,Ulrich F O Luhmann,Ulrich F O Luhmann,Koji M. Nishiguchi,Koji M. Nishiguchi,Jennifer A. E. Williams,John Greenwood,Stephen E. Moss,H Han,Selina A. Azam,Yanai Duran,Scott J Robbie,James W B Bainbridge,James W B Bainbridge,Daniel F P Larkin,Alexander J. Smith,Robin R. Ali +17 more
TL;DR: Results provide proof-of-concept for a strategy to induce in situ regeneration of RPE for the treatment of R PE degeneration in mice by using E2F2 to induce RPE cell replication and thereby regeneration.
Journal ArticleDOI
The pigmented epithelium, a bright partner against photoreceptor degeneration.
TL;DR: The different ancillary roles that the pigmented cells play in the physiology and homeostasis of photoreceptors are examined, linking each one of these processes to the most common hereditary retinal diseases.
Journal ArticleDOI
Optimizing Donor Cellular Dissociation and Subretinal Injection Parameters for Stem Cell-Based Treatments
Brittni A. Scruggs,Chunhua Jiao,Cathryn M. Cranston,Emily E. Kaalberg,Kai Wang,Stephen R. Russell,Luke A Wiley,Robert F. Mullins,Edwin M. Stone,Budd A. Tucker,Elliott H. Sohn +10 more
TL;DR: The effects of commonly used surgical techniques on both host and donor cell viability were evaluated in Yucatan mini‐pigs, and a significant increase in cell viability was found when papain was used for RPC isolation.
Journal ArticleDOI
Juvenile Macular Degenerations.
Pablo Altschwager,Lucia Ambrosio,Emily A Swanson,Anne Moskowitz,Anne Moskowitz,Anne B. Fulton,Anne B. Fulton +6 more
TL;DR: The following 3 common juvenile macular degenerations are reviewed: Stargardt disease, X-linked retinoschisis, and Best vitelliform macular dystrophy.
Book ChapterDOI
Mathematical Models of Pluripotent Stem Cells: At the Dawn of Predictive Regenerative Medicine.
Pınar Pir,Nicolas Le Novère +1 more
TL;DR: This chapter gives an overview of existing mathematical models of pluripotency and cell fate, to illustrate the variety of methods and questions and concludes that current approaches are yet to overcome a number of limitations.
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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