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JCI early table of contents for May 8, 2013
Date:5/8/2013

Gene replacement in pigs ameliorates cystic fibrosis-associated intestinal obstruction

Cystic fibrosis (CF) is caused by mutations in CFTR and is characterized by dysfunction of the lungs, liver, pancreas, and intestines. Approximately 15% of babies with CF are born with an obstruction of the small intestine known as meconium ileus, frequently the first sign of CF. Unlike in humans, meconium ileus occurs in 100% of newborn CF pigs. In this issue of the Journal of Clinical Investigation, Michael Welsh and colleagues at the University of Iowa demonstrate that transgenic expression of normal CFTR in the intestine of CF pigs alleviated meconium ileus. Over time, the pigs still exhibited other manifestations of CF, including liver and lung disease, reduced weight gain, and pancreatic destruction. These findings provide insight into the pathophysiology of CF and indicate that tissue-specific, partial gene replacement can ameliorate intestinal symptoms of CF.

TITLE:
Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs

AUTHOR CONTACT:
Michael Welsh
Howard Hughes Medical Institute, Iowa City, IA, USA
Phone: 319-335-7619; Fax: 319-335-7623; E-mail: michael-welsh@uiowa.edu

View this article at: http://www.jci.org/articles/view/68867?key=d303e72197e1b7e96749


Alzheimer's disease is associated with removal of the synaptic protein ADAM10

Alzheimer's disease is characterized by the accumulation of neurotoxic β-amyloid peptide (Aβ). ADAM10, a protein that resides in the neural synapses, has previously been shown to prevent the formation of Aβ. In this issue of the Journal of Clinical Investigation, Monica Di Luca and colleagues at the University of Milan in Milan, Italy, report that ADAM10 is removed from synapses through asso
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Contact: Jillian Hurst
press_releases@the-jci.org
Journal of Clinical Investigation
Source:Eurekalert

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