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Stem Cell-Based Therapeutics in Ophthalmology: Application Toward the Design of Clinical Trials

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Stem Cell Biology and Regenerative Medicine in Ophthalmology

Part of the book series: Stem Cell Biology and Regenerative Medicine ((STEMCELL))

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Abstract

Stem cell-based therapeutics have been proposed as a technology for restoration of anatomic structure and visual function for retinal degenerative diseases. Success in animal studies and preliminary trials may offer hope for patients afflicted by a variety of retinal degenerations. However, as clinical trials expand and advance to latter phases, it is important to address key study design issues. This chapter discusses the parameters for research into stem cell-based therapeutics. Efficacy endpoints for studies can be defined along objective physiologic, psychofunctional, anatomic, and functional living axes. Pupillometry, electroretinography, and radiologic tools are discussed as objective tools for the assessment of treatment outcome. Additionally, optical coherence tomography(OCT), fundus autofluorescence (FAF), and other imaging tools may be used. Psychofunctional tests may be less reliable among a pediatric population. Finally, improvements in functional living may be reported by patients and assessed by various measures.

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Author information

Authors and Affiliations

  1. Department of Ophthalmology, Columbia University, New York, NY, USA

    Rony Gelman

  2. Department of Ophthalmology & Department of Pathology & Cell Biology, Columbia University, New York, NY, USA

    Stephen H. Tsang

  3. Columbia Stem Cell Initiative, Columbia University Medical Center, New York, NY, USA

    Stephen H. Tsang

Authors
  1. Rony Gelman
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  2. Stephen H. Tsang
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Corresponding author

Correspondence to Rony Gelman .

Editor information

Editors and Affiliations

  1. Edward S. Harkness Eye Institute, Department of Ophthalmology, Columbia University, 160 Fort Washington Avenue, Room 513, New York, 10032, New York, USA

    Stephen H. Tsang

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Gelman, R., Tsang, S.H. (2013). Stem Cell-Based Therapeutics in Ophthalmology: Application Toward the Design of Clinical Trials. In: Tsang, S. (eds) Stem Cell Biology and Regenerative Medicine in Ophthalmology. Stem Cell Biology and Regenerative Medicine. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4614-5493-9_11

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