Nature Biotech: In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency

Plasmid constructs provided by Sangamo BioSciences (SGMO).
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http://www.nature.com/nbt/journal/vaop/ncurrent/full/nbt.3241.html

Surfactant proteins A, B, C, and D are specialized proteins that make up about 5% of the pulmonary surfactant. Surfactant protein B (SP-B) and C (SP-C) are mainly involved in preventing alveolar collapse while surfactant protein A (SP-A) and D (SP-D) play a role in the lung’s immune defense. ABCA3 is a protein that transports surfactant within the alveolar Type II cell, the cell type in the lung that produces pulmonary surfactant. The thyroid transcription factor (TTF1) is a protein that activates surfactant associated genes, among others. Problems with any of these can cause lung damage.

Genome editing using a variety of different nucleases holds great potential to knock out or repair disease-causing genes. An ideal nuclease delivery vehicle is short-lived, does not integrate into the genome and can enter target cells efficiently. These requirements have not yet been achieved simultaneously by any nuclease…