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Editorial

The challenges of developing a gene therapy for amyotrophic lateral sclerosis

, , &
Pages 323-325 | Received 12 Dec 2016, Accepted 24 Jan 2017, Published online: 22 Feb 2017

References

  • Peters OM, Ghasemi M, Brown RH Jr. Emerging mechanisms of molecular pathology in ALS. J Clin Invest. 2015;125:2548.
  • O’Connor DM, Boulis NM. Gene therapy for neurodegenerative diseases. Trends Mol Med. 2015;21:504–512.
  • Picher-Martel V, Valdmanis PN, Gould PV, Julien JP, Dupre N. From animal models to human disease: a genetic approach for personalized medicine in ALS. Acta neuropathologica communications. 2016;4:70.
  • Byrne S, Walsh C, Lynch C, et al. Rate of familial amyotrophic lateral sclerosis: a systematic review and meta-analysis. J Neurol Neurosurg Psychiatry. 2011;82:623–627.
  • Scarrott JM, Herranz-Martin S, Alrafiah AR, et al. Current developments in gene therapy for amyotrophic lateral sclerosis. Expert Opin Biol Ther. 2015;15:935–947.
  • DeJesus-Hernandez M, Mackenzie IR, Boeve BF, et al. Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS. Neuron. 2011;72:245–256.
  • Kenna KP, Van Doormaal PT, Dekker AM, et al. NEK1 variants confer susceptibility to amyotrophic lateral sclerosis. Nat Genet. 2016;48:1037–1042.
  • Golby R, Poirier B, Fabros M, et al. Five-year incidence of amyotrophic lateral sclerosis in British Columbia (2010-2015). Canadian Journal Neurological Sciences Le Journal Canadien Des Sciences Neurologiques. 2016;43(6):791–795.
  • Umoh ME, Fournier C, Li Y, et al. Comparative analysis of C9orf72 and sporadic disease in an ALS clinic population. Neurology. 2016;87:1024–1030.
  • Foust KD, Salazar DL, Likhite S. et al. Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. Mol Ther. 2013;21(12):2148–2159.
  • Stoica L, Todeasa SH, Cabrera GT, et al. Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model. Ann Neurol. 2016;79:687–700.
  • Benkler C, Barhum Y, Ben-Zur T, et al. Multifactorial gene therapy enhancing the glutamate uptake system and reducing oxidative stress delays symptom onset and prolongs survival in the SOD1-G93A ALS mouse model. J Mol Neurosci. 2016;58:46–58.
  • Sances S, Bruijn LI, Chandran S, et al. Modeling ALS with motor neurons derived from human induced pluripotent stem cells. Nat Neurosci. 2016;19:542–553.
  • Yang H, Wang G, Sun H, et al. Species-dependent neuropathology in transgenic SOD1 pigs. Cell Res. 2014;24:464–481.
  • Tu Z, Yang W, Yan S, et al. CRISPR/Cas9: a powerful genetic engineering tool for establishing large animal models of neurodegenerative diseases. Mol Neurodegener. 2015;10:35.
  • Choudhury SR, Hudry E, Maguire CA, et al. Viral vectors for therapy of neurologic diseases. Neuropharmacology. 2016Feb 21. pii: S0028-3908(16)30048-X.
  • Hirano M, Kato S, Kobayashi K, et al. Highly efficient retrograde gene transfer into motor neurons by a lentiviral vector pseudotyped with fusion glycoprotein. Plos One. 2013;8:e75896.
  • Suzuki M, Svendsen CN. Ex vivo gene therapy using human mesenchymal stem cells to deliver growth factors in the skeletal muscle of a familial ALS rat model. Methods Mol Biol. 2016;1382:325–336.
  • Stewart S. Press release: cedars-Sinai receives approval to test novel combined stem cell and gene therapy for ALS patients.Los Angeles, CA: Cedars-Sinai; 2016.
  • Saraiva J, Nobre RJ, De Almeida LP. Gene therapy for the CNS using AAVs: the impact of systemic delivery by AAV9. Journal of controlled release: official journal of the Controlled Release Society; 2016;241:94–109.
  • Federici T, Boulis NM. Gene therapy for amyotrophic lateral sclerosis. Neurobiol Dis. 2012;48:236–242.
  • Mendell JR. Phase I gene transfer clinical trial for spinal muscular atrophy type 1 delivering AVXS-101. In: ClinicalTrials.gov [internet]. Bethesda (MD): National Library of Medicine (US); 2014-2016. 2014. Available from: https://clinicaltrials.gov/ct2/show/record/NCT02122952.
  • Federici T, Riley J, Park J, et al. Preclinical safety validation of a stabilized viral vector direct injection approach to the cervical spinal cord. Clin Transl Sci. 2009;2:165–167.
  • Mazzini L, Gelati M, Profico DC, et al. Human neural stem cell transplantation in ALS: initial results from a phase I trial. J Transl Med. 2015;13:17.

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