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
Stem Cell Therapies in Clinical Trials: Progress and Challenges
TL;DR: Clinical investigations using stem cell products in regenerative medicine are addressing a wide spectrum of conditions using a variety of stem cell types and applications are progressing in trials, some with early benefits to patients.
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
Stem cells: past, present, and future.
TL;DR: A wide variety of possibilities makes this cutting edge therapy a turning point in modern medicine, providing hope for untreatable diseases and challenges that stem cell therapy must overcome to be accepted worldwide.
Journal ArticleDOI
Gene therapy clinical trials worldwide to 2017: An update
Samantha L. Ginn,Anais K. Amaya,Ian E. Alexander,Ian E. Alexander,Michael Edelstein,Mohammad R. Abedi +5 more
TL;DR: This review presents the analysis of clinical trials that, to the best of the knowledge, have been or are being performed worldwide, and discusses key trends since the previous review, namely the use of chimeric antigen receptor T cells for the treatment of cancer and advancements in genome editing technologies.
Journal ArticleDOI
Phase 1 clinical study of an embryonic stem cell-derived retinal pigment epithelium patch in age-related macular degeneration.
Lyndon da Cruz,Kate Fynes,Odysseas Georgiadis,Odysseas Georgiadis,Odysseas Georgiadis,Julie Kerby,Yvonne Hsu-Lin Luo,Yvonne Hsu-Lin Luo,Yvonne Hsu-Lin Luo,Ahmad Ahmado,Amanda J. Vernon,Julie T. Daniels,Britta Nommiste,Shazeen M Hasan,Sakina B Gooljar,Amanda-Jayne F. Carr,Anthony A. Vugler,Conor M. Ramsden,Conor M. Ramsden,Magda Bictash,Mike Fenster,Juliette Steer,Tricia Harbinson,Anna Wilbrey,Adnan Tufail,Adnan Tufail,Gang Feng,Mark Whitlock,Anthony G. Robson,Anthony G. Robson,Graham E. Holder,Graham E. Holder,Mandeep S. Sagoo,Mandeep S. Sagoo,Peter T. Loudon,Paul J. Whiting,Paul J. Whiting,Peter J. Coffey,Peter J. Coffey,Peter J. Coffey +39 more
TL;DR: This work supports the feasibility and safety of hESC-RPE patch transplantation as a regenerative strategy for AMD and presents the preclinical surgical, cell safety and tumorigenicity studies leading to trial approval.
References
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Journal ArticleDOI
The Retinal Pigment Epithelium in Health and Disease
TL;DR: The magnitude of the demands imposed on this single layer of cells in order to execute these tasks, will become apparent to the reader of this review as will the number of clinical disorders that take origin from these cells.
Journal ArticleDOI
Long‐Term Safety and Function of RPE from Human Embryonic Stem Cells in Preclinical Models of Macular Degeneration
Bin Lu,Christopher Malcuit,Shaomei Wang,S.V. Girman,Peter J. Francis,Linda Lemieux,Robert Lanza,R. D. Lund +7 more
TL;DR: Long‐term data showed no gross or microscopic evidence of teratoma/tumor formation after subretinal hESC‐RPE transplantation, suggesting that hESCs could serve as a potentially safe and inexhaustible source of RPE for the efficacious treatment of a range of retinal degenerative diseases.
Journal ArticleDOI
Teratoma formation by human embryonic stem cells: evaluation of essential parameters for future safety studies.
TL;DR: The teratoma assays are considered the gold standard for demonstrating differentiation potential of pluripotent human embryonic stem cells and hold promise as a standard for assessing safety among hESC-derived cell populations intended for therapeutic applications.
Journal ArticleDOI
Identifying the content area for the 51-item National Eye Institute Visual Function Questionnaire: results from focus groups with visually impaired persons.
Carol M. Mangione,Sandra Berry,Karen L. Spritzer,Nancy K. Janz,Ronald Klein,Cynthia Owsley,Paul P. Lee +6 more
TL;DR: An item-generation strategy for a new questionnaire using a standardized focus group method identified content areas and aspects of visual disability that are not included in currently available vision-specific instruments that assess the impact of common eye diseases on visual functioning in every-day life.
Journal ArticleDOI
Human Embryonic Stem Cell–Derived Cells Rescue Visual Function in Dystrophic RCS Rats
Raymond D. Lund,Shaomei Wang,Irina Klimanskaya,Toby Holmes,Rebeca Ramos-Kelsey,Bin Lu,S.V. Girman,N. Bischoff,Yves Sauve,Robert Lanza +9 more
TL;DR: Embryonic stem cells promise to provide a well-characterized and reproducible source of replacement tissue for human clinical studies and batches of RPE derived from NIH-approved hES cells were tested and shown capable of extensive photoreceptor rescue in an animal model of retinal disease.
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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