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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Techniques of Human Embryonic Stem Cell and Induced Pluripotent Stem Cell Derivation.
TL;DR: This review discusses key issues concerning the techniques of pluripotent cell acquisition and reveals other barriers to overcome, such as epigenetic memory, disparity between human and mouse pluripotency, and variable response to differentiation of some iPSC lines.
Journal ArticleDOI
Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy
TL;DR: Current technological limitations and barriers that need to be overcome before gene augmentation therapies for retinal dystrophies can become a viable treatment option for patients are highlighted.
Journal ArticleDOI
Manufacturing Cell Therapies Using Engineered Biomaterials
Amr A. Abdeen,Krishanu Saha +1 more
TL;DR: The use of engineered biomaterials to control human cell manufacturing has the potential to generate manufacturing processes that produce standardized cells with well-defined critical quality attributes appropriate for clinical testing.
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The Future of Regenerative Medicine: Cell Therapy Using Pluripotent Stem Cells and Acellular Therapies Based on Extracellular Vesicles.
Margot Jarrige,Margot Jarrige,Elie Frank,Elie Frank,Elise Herardot,Elise Herardot,Sabrina Martineau,Sabrina Martineau,Annabelle Darle,Annabelle Darle,Manon Benabides,Manon Benabides,Sophie Domingues,Sophie Domingues,Olivier Chose,Olivier Chose,Walter Habeler,Walter Habeler,Judith Lorant,Judith Lorant,Christine Baldeschi,Christine Baldeschi,Cécile Martinat,Cécile Martinat,Christelle Monville,Christelle Monville,Lise Morizur,Lise Morizur,Karim Ben M'Barek,Karim Ben M'Barek +29 more
TL;DR: In this paper, the state of the art of regenerative medicine and its current limitations and challenges are discussed, with particular attention on pluripotent stem cell-derived products to repair organs like the eye, heart, skeletal muscle and skin.
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High-risk corneal allografts: A therapeutic challenge.
TL;DR: The advances in technology and research indicate that wider therapeutic choices for patients may be available to address the worldwide problem of corneal blindness in both "low- risk" and "high-risk" hosts, and developments in regenerative medicine may be able to solve both important short comings of allotransplantation.
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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