Skip to main content
SpringerLink
Log in

Restoring Vision Using Stem Cells and Transplantation

  • Conference paper
  • First Online:
Retinal Degenerative Diseases

Part of the book series: Advances in Experimental Medicine and Biology ((AEMB,volume 1185))

Abstract

The replacement of retinal cells, or the support of surviving retinal neurons, in a degenerated retina presents a significant challenge in the fields of ophthalmology and regenerative medicine. Stem cell-based therapies are being explored as an approach for treating retinal dystrophies, such as retinitis pigmentosa (RP), Stargardt’s disease, and age-related macular degeneration (AMD). This review provides an update on the recent progress made toward the restoration of vision lost to degenerative disease using stem cell-based transplantation strategies and the challenges that need to be overcome. Both retinal pigmented epithelium (RPE) and photoreceptor replacement therapies are discussed.

This is a preview of subscription content, log in via an institution to check access.

Access this chapter

Log in via an institution
Chapter
USD 29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD 299.00
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD 379.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book
USD 379.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Institutional subscriptions

References

  • Bainbridge JWB et al (2015) Long-term effect of gene therapy on Leber’s congenital amaurosis. N Engl J Med 372:1887–1897

    Article  Google Scholar 

  • Barnea-Cramer AO et al (2016) Function of human pluripotent stem cell-derived photoreceptor progenitors in blind mice. Sci Rep 6:1–15

    Article  Google Scholar 

  • Da Cruz L et al (2018) Phase 1 clinical study of an embryonic stem cell-derived retinal pigment epithelium patch in age-related macular degeneration. Nat Biotechnol 36:328–337

    Article  Google Scholar 

  • Gonzalez-Cordero A et al (2017) Recapitulation of human retinal development from human pluripotent stem cells generates transplantable populations of cone photoreceptors. Stem Cell Reports 9:820–837

    Article  Google Scholar 

  • Gonzalez-Cordero A et al (2013) Photoreceptor precursors derived from three-dimensional embryonic stem cell cultures integrate and mature within adult degenerate retina. Nat Biotechnol 31:741–747

    Article  CAS  Google Scholar 

  • Idelson M et al (2009) Directed differentiation of human embryonic stem cells into functional retinal pigment epithelium cells. Cell Stem Cell 5:396–408

    Article  CAS  Google Scholar 

  • Kamao H et al (2014) Characterization of human induced pluripotent stem cell-derived retinal pigment epithelium cell sheets aiming for clinical application. Stem Cell Reports 2:205–218

    Article  CAS  Google Scholar 

  • Kashani AH et al (2018) A bioengineered retinal pigment epithelial monolayer for advanced, dry-age-related macular degeneration. Sci Transl Med 10:1–10

    Article  Google Scholar 

  • Kruczek K et al (2017) Differentiation and transplantation of embryonic stem cell-derived cone photoreceptors into a mouse model of end-stage retinal degeneration. Stem Cell Reports 8:1659–1674

    Article  CAS  Google Scholar 

  • Lamba DA, Gust J, Reh TA (2009) Transplantation of human embryonic stem cell-derived photoreceptors restores some visual function in Crx-deficient mice. Cell Stem Cell 4:73–79

    Article  CAS  Google Scholar 

  • MacLaren RE et al (2006) Retinal repair by transplantation of photoreceptor precursors. Nature 444:203–207

    Article  CAS  Google Scholar 

  • Mandai M et al (2017a) iPSC-derived retina transplants improve vision in rd1 end-stage retinal-degeneration mice. Stem Cell Reports 8:69–83

    Article  Google Scholar 

  • Mandai M et al (2017b) Autologous induced stem-cell-derived retinal cells for macular degeneration. N Engl J Med 376:1038–1046

    Article  CAS  Google Scholar 

  • Nakano T et al (2012) Self-formation of optic cups and storable stratified neural retina from human ESCs. Cell Stem Cell 10:771–785

    Article  CAS  Google Scholar 

  • Ortin-Martinez A et al (2017) A reinterpretation of cell transplantation: GFP transfer from donor to host photoreceptors. Stem Cells 35:932–939

    Article  CAS  Google Scholar 

  • Osakada F et al (2008) Toward the generation of rod and cone photoreceptors from mouse, monkey and human embryonic stem cells. Nat Biotechnol 26:215–224

    Article  CAS  Google Scholar 

  • Ovando-Roche P et al (2017) Harnessing the potential of human pluripotent stem cells and gene editing for the treatment of retinal degeneration. Curr Stem Cell Rep 3:112–123

    Article  CAS  Google Scholar 

  • Pearson RA et al (2012) Restoration of vision after transplantation of photoreceptors. Nature 485:99–103

    Article  CAS  Google Scholar 

  • Pearson RA et al (2016) Donor and host photoreceptors engage in material transfer following transplantation of post-mitotic photoreceptor precursors. Nat Commun 7:1–15

    Google Scholar 

  • Pezzullo L et al (2018) The economic impact of sight loss and blindness in the UK population. BMC Health Serv Res 18:1–13

    Article  Google Scholar 

  • Phillips MJ et al (2012) Blood-derived human iPS cells generate optic vesicle-like structures with the capacity to form retinal laminae and develop synapses. Invest Ophthalmol Vis Sci 53:2007–2019

    Article  Google Scholar 

  • Powell C et al (2016) Zebrafish Muller glia-derived progenitors are multipotent, exhibit proliferative biases and regenerate excess neurons. Sci Rep 6:1–10

    Article  Google Scholar 

  • Ramsden CM et al (2013) Stem cells in retinal regeneration: past, present and future. Development 140:2576–2585

    Article  CAS  Google Scholar 

  • Santos-Ferreira TF et al (2016) Retinal transplantation of photoreceptors results in donor-host cytoplasmic exchange. Nat Commun 7:1–7

    Article  Google Scholar 

  • Schwartz SD et al (2015) 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. Lancet 385:509–516

    Article  Google Scholar 

  • Shirai H et al (2015) Transplantation of human embryonic stem cell-derived retinal tissue in two primate models of retinal degeneration. PNAS 113:E81–E90

    Article  Google Scholar 

  • Singh MS et al (2013) Reversal of end-stage retinal degeneration and restoration of visual function by photoreceptor transplantation. PNAS 110:1101–1106

    Article  CAS  Google Scholar 

  • Welby E et al (2017) Isolation and comparative transcriptome analysis of human fetal and iPSC-derived cone photoreceptor cells. Stem Cell Reports 9:1898–1915

    Article  CAS  Google Scholar 

  • Zhong X et al (2014) Generation of three-dimensional retinal tissue with functional photoreceptors from human iPSCs. Nat Commun 5:1–14

    Google Scholar 

Download references

Acknowledgment

This work was supported by the MRC UK grant MR/M015688/1, GOSHCC, and the NIHR.

Author information

Authors and Affiliations

  1. Stem Cells and Regenerative Medicine Section, University College London Great Ormond Street Institute of Child Health, and NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK

    Elisa Cuevas, Paresh Parmar & Jane C. Sowden

Authors
  1. Elisa Cuevas
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Paresh Parmar
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Jane C. Sowden
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Jane C. Sowden .

Editor information

Editors and Affiliations

  1. Department of Ophthalmology, Duke University, Durham, NC, USA

    Catherine Bowes Rickman

  2. Department of Ophthalmology, University Hospital Zurich, Zurich, Switzerland

    Christian Grimm

  3. Health Sciences Center, University of Oklahoma, Oklahoma City, OK, USA

    Robert E. Anderson

  4. Ophthalmology, University of Florida, Gainesville, FL, USA

    John D. Ash

  5. Beckman Vision Center, University of California, San Francisco, San Fancisco, CA, USA

    Matthew M. LaVail

  6. Department of Ophthalmology, Case Western Reserve University, Cleveland, OH, USA

    Joe G. Hollyfield

Rights and permissions

Reprints and permissions

Copyright information

© 2019 Springer Nature Switzerland AG

About this paper

Check for updates. Verify currency and authenticity via CrossMark

Cite this paper

Cuevas, E., Parmar, P., Sowden, J.C. (2019). Restoring Vision Using Stem Cells and Transplantation. In: Bowes Rickman, C., Grimm, C., Anderson, R., Ash, J., LaVail, M., Hollyfield, J. (eds) Retinal Degenerative Diseases. Advances in Experimental Medicine and Biology, vol 1185. Springer, Cham. https://doi.org/10.1007/978-3-030-27378-1_92

Download citation

Publish with us

Policies and ethics

Search

Navigation