Interesting article: cystic fibrosis pig model

September 28, 2008

This article struck me as interesting. It may prove to be a better

model of cystic fibrosis (caused by CFTR gene deficiency) than the

mouse model. I feel sorry for the piggies, but I hope that they will

help accelerate research to provide better treatments for cystic

fibrosis patients. Maybe this will also be helpful in PSC because the

liver pathology in this new pig model looks very much like human

primary sclerosing cholangitis. In the mouse model, sclerosing

cholangitis only develops after the mice are given colitis. In this

new pig model, the bile-duct inflammation is evident soon after

birth.

____________________________________________

Science 2008 Sep 26;321(5897):1837-41.

Disruption of the CFTR gene produces a model of cystic fibrosis in

newborn pigs.

CS, Stoltz DA, Meyerholz DK, Ostedgaard LS, Rokhlina T, Taft

PJ, Rogan MP, Pezzulo AA, Karp PH, Itani OA, Kabel AC, Wohlford-

Lenane CL, GJ, Hanfland RA, TL, M, Wax D,

CN, Rieke A, Whitworth K, Uc A, Starner TD, Brogden KA, Shilyansky J,

McCray PB Jr, Zabner J, Prather RS, Welsh MJ.

Department of Internal Medicine, Roy J. and Lucille A. Carver College

of Medicine, University of Iowa, Iowa City, IA 52242, USA.

Almost two decades after CFTR was identified as the gene responsible

for cystic fibrosis (CF), we still lack answers to many questions

about the pathogenesis of the disease, and it remains incurable. Mice

with a disrupted CFTR gene have greatly facilitated CF studies, but

the mutant mice do not develop the characteristic manifestations of

human CF, including abnormalities of the pancreas, lung, intestine,

liver, and other organs. Because pigs share many anatomical and

physiological features with humans, we generated pigs with a targeted

disruption of both CFTR alleles. Newborn pigs lacking CFTR exhibited

defective chloride transport and developed meconium ileus, exocrine

pancreatic destruction, and focal biliary cirrhosis, replicating

abnormalities seen in newborn humans with CF. The pig model may

provide opportunities to address persistent questions about CF

pathogenesis and accelerate discovery of strategies for prevention

and treatment. PMID: 18818360.

____________________________________________

Dave

(father of (23); PSC 07/03; UC 08/03)

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