There are many challenges in finding an effective, long-lasting and universal cure for the whole cohort of patients with Duchenne muscular dystrophy (DMD). The discovery of H19 lncRNA as a stabiliser of dystrophin may prove to be the missing link to the success of various rescue therapies proposed for treating DMD.
References
Azzi, S., Abi Habib, W. & Netchine, I. Curr. Opin. Endocrinol. Diabetes Obes. 21, 30–38 (2014).
Zhang, Y., Li, Y., Hu, Q. & Yang, L. Nat. Cell Biol. https://doi.org/10.1038/s41556-020-00595-5 (2020).
Verhaart, I. E. C. & Aartsma-Rus, A. Nat. Rev. Neurol. 15, 373–386 (2019).
Dumont, N. A. et al. Nat. Med. 21, 1455–1463 (2015).
Evano, B. & Tajbakhsh, S. Regen. Med. 3, 24 (2018).
Godfrey, C. et al. Hum. Mol. Genet. 24, 4225–4237 (2015).
Li, C. & Samulski, R. J. Nat. Rev. Genet. 21, 255–272 (2020).
Nance, M. E. et al. Mol. Ther. 27, 1568–1585 (2019).
Kirschner, J. & Lochmüller, H. Handb. Clin. Neurol. 101, 41–46 (2011).
Chang, N. C. et al. Cell Stem Cell 22, 755–768.e6 (2018).
Wakeling, E. L. et al. Nat. Rev. Endocrinol. 13, 105–124 (2017).
Keniry, A. et al. Nat. Cell Biol. 14, 659–665 (2012).
Morgoulis, D. et al. Cell Death Dis. 10, 10 (2019).
Alipoor, B., Parvar, S. N., Sabati, Z., Ghaedi, H. & Ghasemi, H. Mol. Biol. Rep. 47, 6357–6374 (2020).
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M.A.R. is a Founder and CSO of Satellos Bioscience. M.R. declares no competing interests.
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Ritso, M., Rudnicki, M.A. H19 lncRNA to dystrophin’s rescue. Nat Cell Biol 22, 1289–1290 (2020). https://doi.org/10.1038/s41556-020-00598-2
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DOI: https://doi.org/10.1038/s41556-020-00598-2
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