Transplantation of human embryonic stem cell-derived retinal pigment epithelial cells in macular degeneration
Manjit S Mehat,Venki Sundaram,Caterina Ripamonti,Anthony G. Robson,Alexander J. Smith,Shyamanga Borooah,Martha Robinson,Adam N. Rosenthal,William Innes,Richard G. Weleber,Richard W J Lee,Michael D. Crossland,Gary S. Rubin,Baljean Dhillon,David H. W. Steel,Eddy Anglade,Robert Lanza,Robin R. Ali,Michel Michaelides,James W B Bainbridge +19 more
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TLDR
Subretinal hyperpigmentation is consistent with the survival of viable transplanted hESC-derived RPE cells, but may reflect released pigment in their absence, and intervention in early stage of disease should be approached with caution.About:
This article is published in Ophthalmology.The article was published on 2018-06-05 and is currently open access. It has received 158 citations till now. The article focuses on the topics: Macular degeneration & Microperimetry.read more
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Clinical spectrum, genetic complexity and therapeutic approaches for retinal disease caused by ABCA4 mutations
TL;DR: An in depth assessment of the cumulative knowledge of ABCA4-associated retinopathy – clinical manifestations, genetic complexity, pathophysiology as well as current and proposed therapeutic approaches is provided.
Journal ArticleDOI
Next-generation stem cells — ushering in a new era of cell-based therapies
Erin A. Kimbrel,Robert Lanza +1 more
TL;DR: This Review discusses how cell-engineering approaches are enabling the development of next-generation stem cell therapies with improved function, specificity and responsiveness, thereby expanding their applications into areas such as delivering drugs and oncolytic viruses to tumours and promoting tissue repair in various diseases.
Journal ArticleDOI
Transplantation of photoreceptors into the degenerative retina: Current state and future perspectives.
TL;DR: The history and current state of photoreceptor transplantation, the techniques used to assess rescue of visual function, the prerequisites for effective transplantation as well as the main roadblocks, including safety and immune response to the graft, that need to be overcome for successful clinical translation of photorean transplantation approaches are discussed.
Journal ArticleDOI
Stem Cells, Genome Editing, and the Path to Translational Medicine.
Frank Soldner,Rudolf Jaenisch +1 more
TL;DR: How advances in human pluripotent stem cells (hPSCs) coincide with evolutions in genome engineering and genomic technologies to provide realistic opportunities to tackle some of the most devastating complex disorders is explored.
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Photoreceptor cell replacement in macular degeneration and retinitis pigmentosa: A pluripotent stem cell-based approach
TL;DR: The advances that have been made toward generation and characterization of transplantable photoreceptors from PSCs are outlined and the current status of PSC-based preclinical studies for photoreceptor replacement conducted in animal models are summarized.
References
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Disruption of epithelial cell-matrix interactions induces apoptosis
Steven M. Frisch,Hunter Francis +1 more
TL;DR: It is demonstrated that apoptosis was induced by disruption of the interactions between normal epithelial cells and extracellular matrix, and the circumvention of anoikis accompanies the acquisition of anchorage independence or cell motility.
Journal ArticleDOI
A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy
Rando Allikmets,Nanda A. Singh,Hui Sun,Noah F. Shroyer,Amy Hutchinson,Abirami Chidambaram,Bernard Gerrard,Lisa Baird,Dora Stauffer,Andy Peiffer,Amir Rattner,Philip M. Smallwood,Yixin Li,Kent L. Anderson,Richard A. Lewis,Jeremy Nathans,Mark Leppert,Michael Dean,James R. Lupski +18 more
TL;DR: Mutational analysis of ABCR in STGD families revealed a total of 19 different mutations including homozygous mutations in two families with consanguineous parentage, indicating that ABCR is the causal gene of STGD/FFM.
Journal ArticleDOI
Embryonic stem cell trials for macular degeneration: a preliminary report
Steven D. Schwartz,Jean-Pierre Hubschman,Gad Heilwell,Valentina Franco-Cardenas,Carolyn K. Pan,Rosaleen Ostrick,Edmund Mickunas,Irina Klimanskaya,Robert Lanza +8 more
TL;DR: The first description of hESC-derived cells transplanted into human patients with Stargardt's macular dystrophy and dry age-related macular degeneration is provided, with no signs of hyperproliferation, tumorigenicity, ectopic tissue formation, or apparent rejection after 4 months.
Journal ArticleDOI
Autologous Induced Stem-Cell–Derived Retinal Cells for Macular Degeneration
Michiko Mandai,Akira Watanabe,Yasuo Kurimoto,Yasuhiko Hirami,Chikako Morinaga,Takashi Daimon,Masashi Fujihara,Hiroshi Akimaru,Noriko Sakai,Yumiko Shibata,Motoki Terada,Yui Nomiya,Shigeki Tanishima,Masahiro Nakamura,Hiroyuki Kamao,Sunao Sugita,Akishi Onishi,Tomoko Ito,Kanako Fujita,Shin Kawamata,Masahiro J. Go,Chikara Shinohara,Kenichiro Hata,Masanori Sawada,Midori Yamamoto,Sachiko Ohta,Yasuo Ohara,Kenichi Yoshida,Junko Kuwahara,Yuko Kitano,Naoki Amano,Masafumi Umekage,Fumiyo Kitaoka,Azusa Tanaka,Chihiro Okada,Naoko Takasu,Seishi Ogawa,Shinya Yamanaka,Masayo Takahashi +38 more
TL;DR: At 1 year after surgery, the transplanted sheet of retinal pigment epithelial cells remained intact, best corrected visual acuity had not improved or worsened, and cystoid macular edema was present.
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
TL;DR: 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.
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