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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Inherited retinal diseases: Therapeutics, clinical trials and end points-A review.
TL;DR: In this paper, a review of inherited retinal diseases (IRDs) is presented, including macular dystrophies, cone and cone-rod dystrophes, cone dysfunction syndromes, Leber congenital amaurosis, rod-cone dystrophic disorders, rod dysfunction disorders and chorioretinal dystophies.
Journal ArticleDOI
Rare genetic diseases: update on diagnosis, treatment and online resources.
Robert Pogue,Denise P. Cavalcanti,Shreya Shanker,Rosangela Vieira Andrade,Lana Ribeiro Aguiar,Juliana Lott Carvalho,Fabrício F. Costa +6 more
TL;DR: How the collection of various types of information together with the use of new technologies is facilitating diagnosis and, consequently, treatment of rare diseases is discussed.
Journal ArticleDOI
Teratoma: from spontaneous tumors to the pluripotency/malignancy assay
TL;DR: The standardized teratoma assay should be directed more to the assessment of EC/malignant cell features than of differentiated tissues, especially after a unique case of human therapy with neural stem cells was found to lead to malignancy.
Journal ArticleDOI
Chemical Modulation of Cell Fate in Stem Cell Therapeutics and Regenerative Medicine
TL;DR: Recent progress, new insights, and future challenges of the chemical approaches in stem cell biology and regenerative medicine are discussed.
Journal ArticleDOI
HiPSC-derived retinal ganglion cells grow dendritic arbors and functional axons on a tissue-engineered scaffold.
Li Kangjun,Xiufeng Zhong,Sijing Yang,Ziming Luo,Kang Li,Ying Liu,Song Cai,Huai-Yu Gu,Lu Shoutao,Haijun Zhang,Yantao Wei,Jing Zhuang,Yehong Zhuo,Zhigang Fan,Jian Ge +14 more
TL;DR: The generation of retinal ganglion cells from the neural retina and their seeding on a biodegradable poly (lactic-co-glycolic acid) scaffold to create an engineered RGC-scaffold biomaterial is disclosed.
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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