Concepedia

Self-assembled peptide–poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis

Shan Guan, Antje Munder, Silke Hedtfeld, Peter Braubach, Silke Glage, Longgui Zhang, Stefan Lienenklaus, Anja Schultze, Günther Hasenpusch, Wiebke Garrels,

Nature Nanotechnology · 2019 · 132 citations · 36 references

DOI

Concepts

References

36

Principles of nanoparticle design for overcoming biological barriers to drug delivery

Elvin Blanco, Haifa Shen, Mauro Ferrari · Nature Biotechnology · 2015 · 6.7K citations · Full text

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Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis

Seng H. Cheng, Richard J. Gregory, John Marshall et al. · Cell · 1990 · 1.8K citations

, , +12

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

John A. Zuris, David B. Thompson, Yilai Shu et al. · Nature Biotechnology · 2014 · 1.5K citations · Full text

, , +12

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

Gerald Schwank, Bon‐Kyoung Koo, Valentina Sasselli et al. · Cell stem cell · 2013 · 1.4K citations · Full text

, , +16

A functional CFTR assay using primary cystic fibrosis intestinal organoids

Johanna F. Dekkers, Caroline L. Wiegerinck, Hugo R. de Jonge et al. · Nature Medicine · 2013 · 997 citations

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