2015

Singh NN, Lee BM, DiDonato CJ, Singh RN. Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy. Future Med Chem. 2015 Sep;7(13):1793-808.

Heier CR, DiDonato CJ. ECG in neonate mice with spinal muscular atrophy allows assessment of drug efficacy. Front Biosci (Elite Ed). 2015 Jan 1

2014

Keil JM, Seo J, Howell MD, Hsu WH, Singh RN, DiDonato CJ. A short antisense oligonucleotide ameliorates symptoms of severe mouse models of spinal muscular atrophy. Mol Ther Nucleic Acids. 2014 Jul 8;3:e174.oi: 10.1038/mtna.2014.23.

2013

Gogliotti RG, Cardona H, Singh J, Bail S, Emery C, Kuntz N, Jorgensen M, Durens M, Xia B, Barlow C, Heier CR, Plasterer HL, Jacques V, Kiledjian M, Jarecki J, Rusche J, DiDonato CJ. The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models. Hum Mol Genet. 2013.

2012

Gogliotti, R.G., Quinlan, K.A., Barlow, C.B., Heier, C.R., Heckman, C.J., Didonato, C.J. Motor neuron rescue in spinal muscular atrophy mice demonstrates that sensory-motor defects are a consequence, not a cause, of motor neuron dysfunction. J Neurosci. 2012 Mar 14;32(11):3818-29.

2011

Gogliotti RG, Lutz C, Jorgensen M, Huebsch K, Koh S, Didonato CJ. Characterization of a commonly used mouse model of SMA reveals increased seizure susceptibility and heightened fear response in FVB/N mice. Neurobiol Dis. 2011 Jul;43(1):142-51. Epub 2011 Mar 21.

2010

Hammond SM, Gogliotti RG, Rao V, Beauvais A, Kothary R, DiDonato CJ. Mouse survival motor neuron alleles that mimic SMN2 splicing and are inducible rescue embryonic lethality early in development but not late. PLoS One. 2010 Dec 29;5(12)

Heier, C.R., Hampton, T.G., Wang, D., DiDonato C.J. (2010) Development of electrocardiogram intervals during growth of FVB/N neonate mice. BMC Physiol. Aug 24;10(1):16.

Heier, C.R., Satta, R., Lutz, C., DiDonato, C.J. (2010) Arrhythmia and cardiac defects are a feature of spinal muscular atrophy model mice.   Human Molecular Genetics. 2010 Oct 15;19(20):3906-18

Gogliotti RG, Hammond SM, Lutz C, Didonato CJ. Molecular and phenotypic reassessment of an infrequently used mouse model for spinal muscular atrophy. Biochem Biophys Res Commun. 2010 Jan 1;391(1):517-22.