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• MYH10 activation rescues contractile defects in arrhythmogenic cardiomyopathy (ACM).

  García-Quintáns N, Sacristán S, Márquez-López C, Sánchez-Ramos C, Martinez-de-Benito F, Siniscalco D, González-Guerra A, Camafeita E, Roche-Molina M, Lytvyn M, Morera D, Guillen MI, Sanguino MA, Sanz-Rosa D, Martín-Pérez D, Garcia R, Bernal JA. García-Quintáns N, et al. Nat Commun. 2023 Oct 13;14(1):6461. doi: 10.1038/s41467-023-41981-5. Nat Commun. 2023. PMID: 37833253 Free PMC article.
• Adeno-associated virus vector-mediated IL-10 gene delivery prevents type 1 diabetes in NOD mice.

  Goudy K, Song S, Wasserfall C, Zhang YC, Kapturczak M, Muir A, Powers M, Scott-Jorgensen M, Campbell-Thompson M, Crawford JM, Ellis TM, Flotte TR, Atkinson MA. Goudy K, et al. Proc Natl Acad Sci U S A. 2001 Nov 20;98(24):13913-8. doi: 10.1073/pnas.251532298. Proc Natl Acad Sci U S A. 2001. PMID: 11717448 Free PMC article.
• Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using rAAV-mediated gene transfer.

  Kirik D, Georgievska B, Burger C, Winkler C, Muzyczka N, Mandel RJ, Bjorklund A. Kirik D, et al. Proc Natl Acad Sci U S A. 2002 Apr 2;99(7):4708-13. doi: 10.1073/pnas.062047599. Epub 2002 Mar 26. Proc Natl Acad Sci U S A. 2002. PMID: 11917105 Free PMC article.
• AAV-mediated lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene replacement therapy rescues retinal degeneration in rd11 mice.

  Dai X, Han J, Qi Y, Zhang H, Xiang L, Lv J, Li J, Deng WT, Chang B, Hauswirth WW, Pang JJ. Dai X, et al. Invest Ophthalmol Vis Sci. 2014 Mar 20;55(3):1724-34. doi: 10.1167/iovs.13-13654. Invest Ophthalmol Vis Sci. 2014. PMID: 24557352 Free PMC article.
• Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.

  Pang JJ, Dai X, Boye SE, Barone I, Boye SL, Mao S, Everhart D, Dinculescu A, Liu L, Umino Y, Lei B, Chang B, Barlow R, Strettoi E, Hauswirth WW. Pang JJ, et al. Mol Ther. 2011 Feb;19(2):234-42. doi: 10.1038/mt.2010.273. Epub 2010 Dec 7. Mol Ther. 2011. PMID: 21139570 Free PMC article.