Stem Cell Biology and Regenerative Medicine i干细胞生物学和再生医学

Stem Cell Biology and Regenerative Medicine i

干细胞生物学和再生医学

Chapter 1
The Eye as a Target Organ for Stem
Cell Therapy
Mark A. Fields, John Hwang, Jie Gong, Hui Cai, and Lucian V. Del Priore
Abstract Retinal degenerations are a heterogeneous group of disorders that are
characterized by progressive cellular dysfunction, cellular disarray, and eventually
cell death. Early in the course of disease therapeutic intervention consists of pharmaceutical
treatment to prevent cell death or gene therapy to correct the underlying
mutation. Due to the nature of pathologies involving these disorders, particularly in
late stage of disease, cell replacement therapy or electric stimulation of remaining cells
by artificial retinal prosthesis is the only viable option. Stem cell therapies for retinal
degenerative diseases such as age-related macular degeneration (AMD) and retinitis
pigmentosa (RP) are a promising therapeutic option and will require replacement of
lost photoreceptor cells and retinal pigment epithelium (RPE). Current clinical trials
are underway to evaluate the potential of stem cell therapy in humans. The use of
induced pluripotent stem (iPS) cells hold great promise as a potential reservoir of cells
for the treatment of retinal disorders as well as a clinical tool to help understand disease
pathology. Advances in stem cell technology will translate these therapies into viable
clinical options for the treatment of retinal degenerative diseases and other disorders.
Introduction
Retinal degenerations are a heterogeneous group of disorders that are characterized
by progressive cellular dysfunction, cellular disarray, and eventually cell death.
Numerous classification systems exist for these disorders, but no one classification
M.A. Fields ?J. Gong ?L.V. Del Priore (*)
Department of Ophthalmology, Albert Florens Storm Eye Institute, Medical University of
South Carolina, Charleston, SC, USA
e-mail:
J. Hwang
Doheny Eye Institute, Keck School of Medicine, University of Southern California,
Los Angeles, CA, USA
H. Cai
Harkness Eye Institute, Columbia University, New York, NY, USA
S.H. Tsang (ed.), Stem Cell Biology and Regenerative Medicine in Ophthalmology,
Stem Cell Biology and Regenerative Medicine, DOI 10.1007/978-1-4614-5493-9_1,
# Springer Science+Business Media New York 2013