First the abstract and then why I am sending it: QUOTE Science. 2013 Oct 4;342(6154):111-114. Allele-Specific Silencing of Mutant Myh6 Transcripts in Mice Suppresses Hypertrophic Cardiomyopathy. Jiang J, Wakimoto H, Seidman JG, Seidman CE. Source Department of Genetics, Harvard Medical School, Boston, MA 02115, USA. Abstract Dominant mutations in sarcomere proteins such as the myosin heavy chains (MHC) are the leading genetic causes of human hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy. We found that expression of the HCM-causing cardiac MHC gene (Myh6) R403Q mutation in mice can be selectively silenced by an RNA interference (RNAi) cassette delivered by an adeno-associated virus vector. RNAi-transduced MHC403/+ mice developed neither hypertrophy nor myocardial fibrosis, the pathologic manifestations of HCM, for at least 6 months. Because inhibition of HCM was achieved by only a 25% reduction in the levels of the mutant transcripts, we suggest that the variable clinical phenotype in HCM patients reflects allele-specific expression and that partial silencing of mutant transcripts may have therapeutic benefit. PMID: 24092743 [PubMed - as supplied by publisher] END QUOTE As has been mentioned here before, cardiomyopathy tends to be a genetic disorder which is seen more often in ferrets if they have Neural Crest genetic variations, and indeed an increase in the rate of all forms of cardiomyopathy is seen in a very wide range of mammals when they have genetic changes to the Neural Crest. Neural Crest Variations are usually seen with extraneous white markings (though they can be hidden because of having variable expression across generations, and because the telltale markings that appear in many individuals with Neural Crest variations are white so are hidden in marked whites, albinos, and DEWs). Incredibly rare -- but possibly bred once as a failed line by a Scandinavian breeder who moved his operation to China after found guilty of animal abuse -- is LEOPARD Syndrome, a Neural Crest variation that causes hyper-pigmented spots (black spots) on skin and fur and which is associated in studies with poor health including cardiomyopathy. Furthermore, there are several loci (genetic locations) at which such alleles could be located so it is possible for an individual to have cumulative Neural Crest genetic variations. Sadly, many of these types of markings were preferentially bred in ferrets, first in the U.S. beginning with private breeders and one medium sized farm, and more recently they began appearing more in parts of Europe. As it stands this work is not useful at this time for most of us, but it does help clarify for everyone why cardiomyopathy happens and shows vets that there is progress in approaches but at this point what is being looked at is not a treatment for cardiomyopathy that is already present. What is being done here is suppressing the development of cardiomyopathy in the first place. Sukie (not a vet) Ferrets make the world a game. Recommended ferret health links: http://pets.groups.yahoo.com/group/ferrethealth/ http://ferrethealth.org/archive/ http://www.miamiferret.org/ http://www.ferrethealth.msu.edu/ all ferret topics: http://listserv.ferretmailinglist.org/archives/ferret-search.html "All hail the procrastinators for they shall rule the world tomorrow." (2010, Steve Crandall) A nation is as free as the least within it. [Posted in FML 7935]