Concepedia

Gene editing of <i>DNAH11</i> restores normal cilia motility in primary ciliary dyskinesia

Michele Lai, Massimo Pifferi, Andrew Bush, Martina Piras, Angela Michelucci, Maria Di Cicco, Ambra Del Grosso, Paola Quaranta, Chiara Cursi, Elena Tantillo,

Journal of Medical Genetics · 2016 · 66 citations · 38 references

DOI

Abstract

This study demonstrates that gene editing can rescue ciliary beating ex vivo, opening up new avenues for treating PCD.

References

38

Absolute quantification by droplet digital PCR versus analog real-time PCR

Christopher M. Hindson, John R. Chevillet, Hilary Briggs et al. · Nature Methods · 2013 · 1.5K citations · Full text

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Gene Editing of <i>CCR5</i> in Autologous CD4 T Cells of Persons Infected with HIV

Pablo Tebas, David Stein, Winson W. Tang et al. · New England Journal of Medicine · 2014 · 1.3K citations · Full text

A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity

Claudio Mussolino, Robert Morbitzer, Fabienne Lütge et al. · Nucleic Acids Research · 2011 · 710 citations · Full text

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Primary ciliary dyskinesia: a consensus statement on diagnostic and treatment approaches in children

Antonio Barbato, Thomas Frischer, Claudia E. Kuehni et al. · European Respiratory Journal · 2009 · 516 citations · Full text

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Mutations in the <i>DNAH11</i> (axonemal heavy chain dynein type 11) gene cause one form of situs inversus totalis and most likely primary ciliary dyskinesia

Lucia Bartoloni, Jean‐Louis Blouin, Yanzhen Pan et al. · Proceedings of the National Academy of Sciences · 2002 · 359 citations · Full text