For most people, misbehaving mucus causes nothing more than a blocked nose. However, for cystic fibrosis sufferers, overly-thick, sticky mucus is the cause of a constant battle against lung infection. A defective CFTR gene is to blame – the protein it codes for disintegrates before it can reach where it's needed to create normal mucus. But there is hope. By putting a special genetic fragment alongside the human CFTR gene and inserting them into the genome of a healthy mouse, human CFTR protein can occur in the right place. Animals with this genetic modification have high concentrations of the protein in the tips of cells lining the nose (stained orange in the top image), whereas cells in mice without the fragment (bottom image) do not. This raises the possibility of eventually using the gene fragment as a gene therapy for the 60,000 cystic fibrosis sufferers worldwide.
Written by Jan Piotrowski
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