Concepedia

IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy

Qiushi Liang, Fabio Catalano, Eva C. Vlaar, Joon M. Pijnenburg, Merel Stok, Yvette van Helsdingen, Arnold G. Vulto, Ans T. van der Ploeg, Niek P. van Til, W.W.M. Pim Pijnappel

Molecular Therapy — Methods & Clinical Development · 2022 · 28 citations · 93 references

DOIFull text

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Concepts

Abstract

as a candidate transgene for future clinical development of HSPC-LVGT for Pompe disease.

References

93

Copper-Zinc Superoxide Dismutase (SOD1) Is Released by Microglial Cells and Confers Neuroprotection against 6-OHDA Neurotoxicity

Elisabetta Polazzi, Ilaria Mengoni, Marco Caprini et al. · Neurosignals · 2012 · 9K citations · Full text

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Microglia: active sensor and versatile effector cells in the normal and pathologic brain

Uwe‐Karsten Hanisch, Helmut Kettenmann · Nature Neuroscience · 2007 · 3.7K citations

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A Third-Generation Lentivirus Vector with a Conditional Packaging System

Tom Dull, Romain Zufferey, Michael T. Kelly et al. · Journal of Virology · 1998 · 3.4K citations · Full text

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Self-Inactivating Lentivirus Vector for Safe and Efficient In Vivo Gene Delivery

Romain Zufferey, Thomas J. Dull, Ronald J. Mandel et al. · Journal of Virology · 1998 · 1.8K citations · Full text

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Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Nathalie Cartier, Salima Hacein‐Bey‐Abina, Cynthia C. Bartholomae et al. · Science · 2009 · 1.5K citations · Full text

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