Jax Labs eBook - 30

IMPACTFUL SOLUTIONS CREATE INNOVATIVE SCIENCE
F.

% Ejection Fraction

A.

root ganglia (Figure 3). Symptomatic mice are heterozygous
Cre-positive mice, compound heterozygous for the frataxin
conditional and the null alleles.
CONCLUSIONS

Tg-Ckmm-cre
Conditional KO

Wild type

Figure 3. Cardiac Specific Conditional Model Cardiac Readouts
A: The cardiac-specific conditional model (Tg-Ckmm-cre Conditional KO) exhibits
a decrease in the percent ejection fraction over the wild type controls, which
demonstrates a decrease in cardiac function. F: Histological images (H&E) showing
dilated myopathy in mutant hearts.

model include body weight loss and mortality, cardiomyopathy
consisting of reduced ejection fraction and fractional shortening,
and increased left ventricular mass (Figure 2). Mutant mice
reach their peak body weight at 60 days of age and have a mean
survival of 86 ± 4.9 days. The phenotype is consistent with that
observed in the Puccio model.
The other model created at The Jackson Laboratory is a
neuronal-specific model using a Parvalbumin cre, designated
as B6(Cg)-Pvalbtm1(cre)Arbr Fxnem2Lutzy Fxnem2.1Lutzy/J (029721). This
model is created via the selective ablation of frataxin in
parvalbumin-expressing tissues, including brain interneurons
and proprioceptive afferent sensory neurons in the dorsal

30 | GENengnews.com

While current transgenic lines can be useful for measuring
increases in the human frataxin protein expression, they do
not exhibit robust neurological phenotypes. The new models
provided by The Jackson Laboratory include next-generation
YG8SR mice with allelic series of 400, 800, and 1000 repeats,
which are now being characterized. The Fxn-Mck-cre (also
referred to as MCK) CKO mice with conditional deletion of frataxin
in the myocardium and the Fxn-Pvalb CKO which exhibit body
weight loss and mortality associated with cardiomyopathy and
neurological phenotypes, respectively, are beneficial in exploring
protein replacement and gene therapy. These models are widely
distributed by and used in in vivo efficacy studies through the
In Vivo Pharmacology Services at The Jackson Laboratory. nn



https://www.jax.org/strain/029721 http://www.GENengnews.com

Jax Labs eBook

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