Concepedia

Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells

Makoto Yoshimitsu, Koji Higuchi, Shobha Ramsubir, Takahiro Nonaka, Vanessa I. Rasaiah, Christopher Siatskas, S-B Liang, Gary J. Murray, Roscoe O. Brady, Jeffrey A. Medin

Gene Therapy · 2006 · 55 citations · 43 references

DOI

Concepts

References

43

The metabolic and molecular bases of inherited disease

Ying-Tsong Chen · Medical Entomology and Zoology · 2001 · 6.9K citations

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<i>LMO2</i> -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

S. Hacein-Bey-Abina, Christof von Kalle, Manfred Schmidt et al. · Science · 2003 · 3.6K citations

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HIV-1 Integration in the Human Genome Favors Active Genes and Local Hotspots

A. R. W. Schröder, Paul Shinn, Huaming Chen et al. · Cell · 2002 · 1.8K citations · Full text

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Safety and Efficacy of Recombinant Human α-Galactosidase A Replacement Therapy in Fabry's Disease

Christine M. Eng, Nathalie Guffon, William R. Wilcox et al. · New England Journal of Medicine · 2001 · 1.5K citations · Full text

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Enzyme Replacement Therapy in Fabry Disease

Raphael Schiffmann, Jeffrey B. Kopp, Howard A. Austin et al. · JAMA · 2001 · 1.3K citations

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