Cell-Based Therapy for Retinal Disease: The New Frontier

Research output: Chapter in Book/Report/Conference proceedingChapter

3 Scopus citations


The availability of noninvasive high-resolution imaging technology, the immune-suppressive nature of the subretinal space, and the existence of surgical techniques that permit transplantation surgery to be a safe procedure all render the eye an ideal organ in which to begin cell-based therapy in the central nervous system. A number of early stage clinical trials are underway to assess the safety and feasibility of cell-based therapy for retinal blindness. Cell-based therapy using embryonic stem cell-derived differentiated cells (e.g., retinal pigment epithelium (RPE)), neural progenitor cells, photoreceptor precursors, and bone marrow-derived hematopoietic stem/progenitor cells has demonstrated successful rescue and/or replacement in preclinical models of human retinal degenerative disease. Additional research is needed to identify the mechanisms that control synapse formation/disjunction (to improve photoreceptor transplant efficacy), to identify factors that limit RPE survival in areas of geographic atrophy (to improve RPE transplant efficacy in eyes with age-related macular degeneration), and to identify factors that regulate immune surveillance of the subretinal space (to improve long-term photoreceptor and RPE transplant survival).

Original languageEnglish (US)
Title of host publicationMethods in Molecular Biology
PublisherHumana Press Inc.
Number of pages15
StatePublished - Jan 1 2019

Publication series

NameMethods in Molecular Biology
ISSN (Print)1064-3745

All Science Journal Classification (ASJC) codes

  • Molecular Biology
  • Genetics


  • Bone marrow-derived hematopoietic stem/progenitor cells
  • Cell-based therapy
  • Clinical trials
  • Embryonic stem cell-derived differentiated cells
  • Neural progenitor cells
  • Photoreceptor precursors
  • Retinal blindness

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  • Cite this

    Zarbin, M. (2019). Cell-Based Therapy for Retinal Disease: The New Frontier. In Methods in Molecular Biology (pp. 367-381). (Methods in Molecular Biology; Vol. 1834). Humana Press Inc.. https://doi.org/10.1007/978-1-4939-8669-9_23