Cure SMA Funding Leads to 16 New Research Publications in 2014

An important goal of our research funding strategy is to share scientific findings with the broader scientific community. Scientists who receive Cure SMA funding often publish their findings in peer-reviewed journals. This means that other scientists can learn from their results, which will pay dividends across the wider landscape of SMA research—allowing us to multiply the impact of our funding.

In 2014, Cure SMA-funded research led to the publishing of sixteen journal articles. Journal publishing is competitive, and only the best and most intriguing results are published. Congratulations to all these authors on their accomplishment. Thank you for helping us move SMA research forward.

2014 Cure SMA-Funded Journal Articles

  1. SMNis essential for the biogenesis of U7 small nuclear ribonucleoprotein and 3′-end formation of histone mRNAs. Tisdale S, Lotti F, Saieva L, Van Meerbeke JP, Crawford TO, Sumner CJ, Mentis GZ, Pellizzoni L. Cell Rep. 2013 Dec 12;5(5):1187-95. doi: 10.1016/j.celrep.2013.11.012.
  2. Requirement of enhanced Survival Motoneuron protein imposed during neuromuscular junction maturation. Kariya S, Obis T, Garone C, Akay T, Sera F, Iwata S, Homma S, Monani UR. J Clin Invest. 2014 Feb 3;124(2):785-800. doi: 10.1172/JCI72017. Epub 2014 Jan 27.
  3. The Smn independent beneficial effects of trichostatin A on an intermediate mouse model of spinal muscular atrophy. Liu H, Yazdani A, Murray LM, Beauvais A, Kothary R. PLoS One. 2014 Jul 1;9(7):e101225. doi: 10.1371/journal.pone.0101225. eCollection 2014.
  4. Autophagy dysregulation in cell culture and animals models of spinal muscular atrophy. Custer SK, Androphy EJ. Mol Cell Neurosci. 2014 Jul;61:133-40. doi: 10.1016/j.mcn.2014.06.006. Epub 2014 Jun 28.
  5. Survival motor neuron protein deficiency impairs myotube formation by altering myogenic gene expression and focal adhesion dynamics. Bricceno KV, Martinez T, Leikina E, Duguez S, Partridge TA, Chernomordik LV, Fischbeck KH, Sumner CJ, Burnett BG. Hum Mol Genet. 2014 Sep 15;23(18):4745-57. doi: 10.1093/hmg/ddu189. Epub 2014 Apr 23.
  6. The effect of diet on the protective action of D156844 observed in spinal muscular atrophy mice. Butchbach ME, Singh J, Gurney ME, Burghes AH. Exp Neurol. 2014 Jun;256:1-6. doi: 10.1016/j.expneurol.2014.03.005. Epub 2014 Mar 25.
  7. Assays for the identification and prioritization of drug candidates for spinal muscular atrophy. Cherry JJ, Kobayashi DT, Lynes MM, Naryshkin NN, Tiziano FD, Zaworski PG, Rubin LL, Jarecki J. Assay Drug Dev Technol. 2014 Aug;12(6):315-41. doi: 10.1089/adt.2014.587.
  8. Molecular mechanisms and animal models of spinal muscular atrophy. Edens BM, Ajroud-Driss S, Ma L, Ma YC. Biochim Biophys Acta. 2014 Aug 1. pii: S0925-4439(14)00244-0. doi: 10.1016/j.bbadis.2014.07.024.
  9. SMN regulates axonal local translation via miR-183/mTOR pathway. Kye MJ, Niederst ED, Wertz MH, Gonçalves ID, Akten B, Dover KZ, Peters M, Riessland M, Neveu P, Wirth B, Kosik KS, Sardi SP, Monani UR, Passini MA, Sahin M. Hum Mol Genet. 2014 Jul 4. pii: ddu350.
  10. A short antisense oligonucleotide ameliorates symptoms of severe mouse models of spinal muscular atrophy. Keil JM, Seo J, Howell MD, Hsu WH, Singh RN, DiDonato CJ. Mol Ther Nucleic Acids 2014 Jul 8;3:e174. doi: 10.1038/mtna.2014.23.
  11. Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept. Arnold WD, Porensky PN, McGovern VL, Iyer CC, Duque S, Li X, Meyer K, Schmelzer L, Kaspar BK, Kolb SJ, Kissel JT, Burghes AH. Ann Clin Transl Neurol. 2014 Jan 1;1(1):34-44.
  12. Dissection of the transversus abdominis muscle for whole-mount neuromuscular junction analysis. Murray L, Gillingwater TH, Kothary R. J Vis Exp. 2014 Jan 11;(83):e51162. doi: 10.3791/51162.
  13. Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA – a dose response study in mice and nonhuman primates. Meyer K, Ferraiuolo L, Schmelzer L, Braun L, McGovern V, Likhite S, Michels O, Govoni A, Fitzgerald J, Morales P, Foust KD, Mendell JR, Burghes AH, Kaspar BK. Mol Ther. 2014 Oct 31. doi: 10.1038/mt.2014.210.
  14. SnapShot-Seq: a method for extracting genome-wide, in vivo mRNA dynamics from a single total RNA sample. Gray JM, Harmin DA, Boswell SA, Cloonan N, Mullen TE, Ling JJ, Miller N, Kuersten S, Ma YC, McCarroll SA, Grimmond SM, Springer M. PLoS One. 2014 Feb 26;9(2):e89673. doi: 10.1371/journal.pone.0089673.
  15. Respiratory muscle function in infants with spinal muscular atrophy type I. Finkel RS, Weiner DJ, Mayer OH, McDonough JM, Panitch HB. Pediatr Pulmonol. 2014 Apr 29. doi: 10.1002/ppul.22997.
  16. Neurodegeneration in spinal muscular atrophy: from disease phenotype and animal models to therapeutic strategies and beyond. Monani UR, De Vivo DC. Future Neurol. 2014 Jan 1;9(1):49-65.

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