Concepedia

Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice

Don Clarke, Yewande Pearse, Shih‐hsin Kan, Steven Q. Le, Valentina Sanghez, Jonathan D. Cooper, Patricia Dickson, Michelina Iacovino

Molecular Therapy — Methods & Clinical Development · 2018 · 16 citations · 39 references

DOIFull text

Open access

Concepts

Abstract

mice. Our findings suggest that genetically engineered iNSCs could potentially be used to deliver enzymes and treat MPS IIIB.

References

39

Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors

Kazutoshi Takahashi, Shinya Yamanaka · Cell · 2006 · 26.5K citations · Full text

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Anti-Vascular Endothelial Growth Factors Protect Retinal Pigment Epithelium Cells Against Oxidation by Modulating Nitric Oxide Release and Autophagy

Stefano De Cillà, Serena Farruggio, Stela Vujosevic et al. · Cellular Physiology and Biochemistry · 2017 · 5.1K citations · Full text

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Autophagy: cellular and molecular mechanisms

D.R. Glick, Sandra Barth, Kay F. Macleod · The Journal of Pathology · 2010 · 3.8K citations · Full text

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Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's Disease

Norman W. Barton, Roscoe O. Brady, James M. Dambrosia et al. · New England Journal of Medicine · 1991 · 1.3K citations · Full text

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Enzyme-Replacement Therapy in Mucopolysaccharidosis I

Emil Kakkis, Joseph Muenzer, George E. Tiller et al. · New England Journal of Medicine · 2001 · 675 citations · Full text

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