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
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
Citations
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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
Development of the 25-item National Eye Institute Visual Function Questionnaire.
TL;DR: Preliminary analyses indicate that the psychometric properties of the NEI VFQ-25 are robust for the eye conditions studied; this suggests that the measure will provide reproducible and valid data when used across multiple conditions of varying severity.
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
Development of the 25-item National Eye Institute visual function questionnaire
TL;DR: Preliminary analyses indicate that the psychometric properties of the NEI VFQ-25 are robust for the eye conditions studied; this suggests that the measure will provide reproducible and valid data when used across multiple conditions of varying severity.
Journal ArticleDOI
Clinical risk factors for age-related macular degeneration: a systematic review and meta-analysis
Usha Chakravarthy,Tien Yin Wong,Tien Yin Wong,Astrid E. Fletcher,Elisabeth Piault,Christopher M. Evans,Gergana Zlateva,Ronald Buggage,Andreas M. Pleil,Paul Mitchell +9 more
TL;DR: Increasing age, current cigarette smoking, previous cataract surgery, and a family history of AMD showed strong and consistent associations with late AMD.
Journal ArticleDOI
Human embryonic stem cell lines derived from single blastomeres
TL;DR: A series of ten separate experiments demonstrating that hES cells can be derived from single blastomeres are reported, which would address the ethical concerns of many, and allow the generation of matched tissue for children and siblings born from transferred PGD embryos.
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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