Concepedia

Erythroid-Progenitor-Targeted Gene Therapy Using Bifunctional TFR1 Ligand-Peptides in Human Erythropoietic Protoporphyria

Arienne Mirmiran, Caroline Schmitt, Thibaud Lefèbvre, Hana Manceau, Raêd Daher, Vincent Oustric, Antoine Poli, J Lacapère, Boualem Moulouel, Hervé Puy,

The American Journal of Human Genetics · 2019 · 27 citations · 14 references

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14

Renal clearance of quantum dots

Hak Soo Choi, Wenhao Liu, Preeti Misra et al. · Nature Biotechnology · 2007 · 4.4K citations · Full text

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Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Eugenio Mercuri, Basil T. Darras, Claudia A. Chiriboga et al. · New England Journal of Medicine · 2018 · 1.4K citations · Full text

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Receptor mediated uptake of peptides that bind the human transferrin receptor

Jae Hoon Lee, Jeffrey A. Engler, James F. Collawn et al. · European Journal of Biochemistry · 2001 · 278 citations · Full text

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Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

Maja M. Janas, Mark K. Schlegel, Carole E. Harbison et al. · Nature Communications · 2018 · 266 citations · Full text

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The penetrance of dominant erythropoietic protoporphyria is modulated by expression of wildtype FECH

Laurent Gouya, Hervé Puy, Anne-Marie Robréau et al. · Nature Genetics · 2001 · 264 citations

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