Cure SMA Funding Leads to 18 New Research Publications in 2015

An important goal of our research funding strategy is to share scientific findings with the broader scientific community. To help accomplish this goal, scientists who receive Cure SMA funding often publish their findings in peer-reviewed journals. Published articles allow the experiments and results to be reviewed and vetted by other scientists, who may then use these as the basis for further experiments—multiplying the impact of our funding.

In 2015, Cure SMA-funded research led to the publishing of 18 journal articles, by researchers from 15 different institutions. 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.

2015 Cure SMA-Funded Journal Articles

  1. The SMN structure reveals its crucial role in snRNP assembly. Seng CO, Magee C, Young PJ, Lorson CL, Allen JP. Hum Mol Genet. 2015 Jan 5. pii: ddu734.
  2. ECG in neonate mice with spinal muscular atrophy allows assessment of drug efficacy. Heier CR, DiDonato CJ. Front Biosci (Elite Ed). 2015 Jan 1;7:122-33.
  3. Assessing the Needs of the SMA Population: Survey Results of Health Care Providers and Families. Halanski MA, Patterson KG, Sund SA, Makholm LM, Schroth MK. SAGE Open. October-December 2014: 1–5.
  4. Astrocytes influence the severity of spinal muscular atrophy. Rindt H, Feng Z, Mazzasette C, Glascock JJ, Valdivia D, Pyles N, Crawford TO, Swoboda KJ, Patitucci TN, Ebert AD, Sumner CJ, Ko CP, Lorson CL.
  5. Molecular mechanisms and animal models of spinal muscular atrophy. Edens BM, Ajroud-Driss S, Ma L, Ma YC. Biochim Biophys Acta. 2015 Apr;1852(4):685-92.
  6. Non-aggregating tau phosphorylation by cyclin-dependent kinase 5 contributes to motor neuron degeneration in spinal muscular atrophy. Miller N, Feng Z, Edens BM, Yang B, Shi H, Sze CC, Hong BT, Su SC, Cantu JA, Topczewski J, Crawford TO, Ko CP, Sumner CJ, Ma L, Ma YC. J Neurosci. 2015 Apr 15;35(15):6038-50.
  7. 209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7-9 November 2014, Heemskerk, The Netherlands. Finkel R, Bertini E, Muntoni F, Mercuri E; ENMC SMA Workshop Study Group. Neuromuscul Disord. 2015 Apr 28. pii: S0960-8966(15)00134-0.
  8. The effect of the DcpS inhibitor D156844 on the protective action of follistatin in mice with spinal muscular atrophy. Harris AW, Butchbach ME. Neuromuscul Disord. 2015 May 27. pii: S0960-8966(15)00159-5.
  9. DcpS is a transcript-specific modulator of RNA in mammalian cells. Zhou M1, Bail S1, Plasterer HL2, Rusche J2, Kiledjian M3. RNA. 2015 Jul;21(7):1306-12.
  10. Transcriptional profiling of differentially vulnerable motor neurons at pre-symptomatic stage in the Smn (2b/-) mouse model of spinal muscular atrophy. Murray LM, Beauvais A, Gibeault S, Courtney NL, Kothary R. Acta Neuropathol Commun. 2015 Sep 15;3:55.
  11. Responses to Fasting and Glucose Loading in a Cohort of Well Children with Spinal Muscular Atrophy Type II. Davis RH, Miller EA, Zhang RZ, Swoboda KJ. J Pediatr. 2015 Oct 7. pii: S0022-3476(15)01027-6.
  12. How far away is spinal muscular atrophy gene therapy? Gillingwater TH, Murray LM. Expert Rev Neurother. 2015 Sep;15(9):965-8.
  13. Electrophysiological Motor Unit Number Estimation (MUNE) Measuring Compound Muscle Action Potential (CMAP) in Mouse Hindlimb Muscles. Arnold WD, Sheth KA, Wier CG, Kissel JT, Burghes AH, Kolb SJ. J Vis Exp. 2015 Sep 25;(103)
  14. Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy. Singh NN, Lee BM, DiDonato CJ, Singh RN. Future Med Chem. 2015 Sep;7(13):1793-808
  15. Understanding the experiences and needs of individuals with Spinal Muscular Atrophy and their parents: a qualitative study. Qian Y, McGraw S, Henne J, Jarecki J, Hobby K, Yeh WS. BMC Neurol. 2015 Oct 24;15(1):217.
  16. Developing therapies for spinal muscular atrophy. Wertz MH, Sahin M. Ann N Y Acad Sci. 2015 Jul 14
  17. SMN deficiency does not induce oxidative stress in SMA iPSC-derived astrocytes or motor neurons. Patitucci TN, Ebert AD. Hum Mol Genet. 2015 Dec 7. pii: ddv489. [Epub ahead of print]
  18. A novel role for CARM1 in promoting nonsense-mediated mRNA decay: potential implications for spinal muscular atrophy. Sanchez G, Bondy-Chorney E, Laframboise J, Paris G, Didillon A, Jasmin BJ, Côté J. Nucleic Acids Res. 2015 Dec 9. pii: gkv1334. [Epub ahead of print]

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