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Review
. 2014 Nov 13;5(4):a017434.
doi: 10.1101/cshperspect.a017434.

Gene therapy using stem cells

Erin R Burnight  1 , Luke A Wiley  1 , Robert F Mullins  1 , Edwin M Stone  2 , Budd A Tucker  1
Affiliations
Review

Gene therapy using stem cells

Erin R Burnight et al. Cold Spring Harb Perspect Med. .

Abstract

Viral-mediated gene augmentation therapy has recently shown success in restoring vision to patients with retinal degenerative disorders. Key to this success was the availability of animal models that accurately presented the human phenotype to test preclinical efficacy and safety. These exciting studies support the use of gene therapy in the treatment of devastating retinal degenerative diseases. In some cases, however, in vivo gene therapy for retinal degeneration would not be effective because the cell types targeted are no longer present. The development of somatic cell reprogramming methods provides an attractive source of autologous cells for transplantation and treatment of retinal degenerative disease. This article explores the development of gene therapy and patient-derived stem cells for the purpose of restoring vision to individuals suffering from inherited retinal degenerations.

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Figures

Figure 1.
Figure 1.
The transgenic pig carrying a human Pro23His rhodopsin transgene displays drastically accelerated retinal degeneration. A–B: Histological analysis of both wild-type (A) and Pro23His (B) pig retinal tissue at 6 mo of age. C–D: Cytochemical analysis of both wild-type (C) and Pro23His (D) pig retinal tissue at 6 mo of age, using wheat germ agglutinin (WGA, red) and DAPI (blue).
Figure 2.
Figure 2.
Schematic diagram depicting a molecular toolbox for exogenous replacement of genes. Genes of various sizes requiring various levels of expression and cell type selectivity.
Figure 3.
Figure 3.
Schematic diagram depicting the proposed pipeline from gene discovery to treatment of inherited retinal degenerative disease.
See this image and copyright information in PMC

References

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