Affiliation: University of Chicago
Country: USA


  1. Du X, Wei C, Hejazi Pastor D, Rao E, Li Y, Grasselli G, et al. α1ACT Is Essential for Survival and Early Cerebellar Programming in a Critical Neonatal Window. Neuron. 2019;: pubmed publisher
    ..Finally, we show that bicistronic expression may be common to the voltage-gated calcium channel (VGCC) gene family and may help explain complex genetic syndromes. ..
  2. Pastor P, Du X, Fazal S, Davies A, Gomez C. Targeting the CACNA1A IRES as a Treatment for Spinocerebellar Ataxia Type 6. Cerebellum. 2018;17:72-77 pubmed publisher
    ..These studies provide the proof of principle that a therapy directed at selectively preventing α1ACT expression could be used to treat SCA6. ..
  3. Du X, GOMEZ C. Spinocerebellar [corrected] Ataxia Type 6: Molecular Mechanisms and Calcium Channel Genetics. Adv Exp Med Biol. 2018;1049:147-173 pubmed publisher
    ..More importantly, the IRES, as the translational control element of ?1ACT, provides a potential therapeutic target for the treatment of SCA6. ..
  4. Karginov T, Pastor D, Semler B, Gomez C. Mammalian Polycistronic mRNAs and Disease. Trends Genet. 2017;33:129-142 pubmed publisher
    ..Identification of additional polycistronic genes may provide new insights into disease therapy and allow for new discoveries of both translational and disease mechanisms. ..
  5. Miyazaki Y, Du X, Muramatsu S, Gomez C. An miRNA-mediated therapy for SCA6 blocks IRES-driven translation of the CACNA1A second cistron. Sci Transl Med. 2016;8:347ra94 pubmed publisher