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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The role of autophagy in age-related macular degeneration.
TL;DR: It is concluded that SQSTM1/p62 is a good marker for impaired autophagy and Hsp70 upregulation could be used as a therapeutic target against retinal pigment epithelium (RPE) degeneration and in the development novel treatments for AMD.
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The contribution of human/non-human animal chimeras to stem cell research.
Sonya Levine,Laura Grabel +1 more
TL;DR: The essential role human/non-human animal chimeras have played in pluripotent stem cell research is discussed, from gaining a better understanding of human biology to providing a stepping stone to human disease treatments.
Journal ArticleDOI
Mapping the dense scotoma and its enlargement in stargardt disease.
TL;DR: Microperimetry is necessary for mapping the scotoma in patients with Stargardt disease, because current imaging is not adequate and standardized grid testing plus a standardized procedure for refining the border of the denseScotoma should allow more precise testing and longitudinal assessment of enlargement rates.
Journal ArticleDOI
Poly(trimethylene carbonate) as an elastic biodegradable film for human embryonic stem cell-derived retinal pigment epithelial cells.
Anni Sorkio,Suvi Haimi,Suvi Haimi,Vincent Verdoold,Kati Juuti-Uusitalo,Dirk W. Grijpma,Dirk W. Grijpma,Heli Skottman +7 more
TL;DR: Flexible and elastic biodegradable PTMC films show potential for retinal tissue engineering applications and have close resemblance to those of the native Bruch's membrane.
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Special Commentary: Early Clinical Development of Cell Replacement Therapy: Considerations for the National Eye Institute Audacious Goals Initiative
Leonard A. Levin,Leonard A. Levin,Joan W. Miller,Donald J. Zack,Martin Friedlander,Lois E.H. Smith +5 more
TL;DR: The Audacious Goals Initiative (AGI) aims to restore vision through the regeneration of neurons and neural connections in the eye and visual system with a particular emphasis on diseases amenable to regenerative medicine and strategies to deal with barriers to progess.
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.
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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