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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A minimally invasive adjustable-depth blunt injector for delivery of pharmaceuticals into the posterior pole.
Ygal Rotenstreich,Adi Tzameret,Adi Tzameret,Sapir E. Kalish,Sapir E. Kalish,Ettel Bubis,Ettel Bubis,Michael Belkin,Michael Belkin,Iris Moroz,Mordechai Rosner,Mordechai Rosner,Itay Levy,Shlomo Margel,Ifat Sher,Ifat Sher +15 more
TL;DR: To investigate the feasibility and safety of a novel minimally invasive adjustable‐depth blunt injector for pharmaceuticals delivery into the posterior segment.
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Novel Strategies for the Improvement of Stem Cells’ Transplantation in Degenerative Retinal Diseases
Simona Delia Nicoară,Sergiu Șușman,Oana Tudoran,Otilia Barbos,Gabriela Cherecheș,Simion Aștilean,Monica Potara,Olga Sorițău +7 more
TL;DR: The main advances in the field of Retinal transplantology and applications of nanotechnology in the treatment of retinal diseases are explored, the challenges are discussed, and new therapeutic approaches in retinal transplantation are suggested.
Journal ArticleDOI
Epiretinal membrane in a subject after transvitreal delivery of palucorcel (CNTO 2476).
TL;DR: The development of an ERM containing both subject (self) cells and palucorcel suggests that paluc orcel egress in the vitreal cavity after retinotomy may contribute to ERM formation and RD and that an alternative delivery method will be required before further studies are conducted.
Journal ArticleDOI
Recent theranostic paradigms for the management of Age-related macular degeneration
TL;DR: The purpose of this review is to summarize the recent advances in the management and treatment stratagems pertaining to AMD viz. molecular targeting, stem cell therapy, nanotechnology and exosomes with special reference to newer technologies like artificial intelligence and 3D printing.
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Living with Stargardt disease: insights from patients and their parents.
Aude Roborel de Climens,Béatrice Tugaut,Carla Dias Barbosa,Ronald Buggage,Catherine Brun-Strang +4 more
TL;DR: This research provides an overview of symptoms experienced by patients with STGD and highlights the dramatic impact these have on patients’ lives, allowing the identification of concepts of importance when evaluating new therapeutic options for STGD.
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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