Alex A Mireault
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Explore the profile of Alex A Mireault including associated specialties, affiliations and a list of published articles.
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Articles
8
Citations
1298
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0
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Recent Articles
1.
Zhang Y, Nishiyama T, Li H, Huang J, Atmanli A, Sanchez-Ortiz E, et al.
Mol Ther Methods Clin Dev
. 2021 Sep;
22:122-132.
PMID: 34485599
Duchenne muscular dystrophy (DMD), caused by mutations in the X-linked dystrophin gene, is a lethal neuromuscular disease. Correction of DMD mutations in animal models has been achieved by CRISPR/Cas9 genome...
2.
Min Y, Chemello F, Li H, Rodriguez-Caycedo C, Sanchez-Ortiz E, Mireault A, et al.
Mol Ther
. 2020 Sep;
28(9):2044-2055.
PMID: 32892813
Duchenne muscular dystrophy (DMD), one of the most common neuromuscular disorders of children, is caused by the absence of dystrophin protein in striated muscle. Deletions of exons 43, 45, and...
3.
Zhang Y, Li H, Min Y, Sanchez-Ortiz E, Huang J, Mireault A, et al.
Sci Adv
. 2020 Mar;
6(8):eaay6812.
PMID: 32128412
Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by mutations in the dystrophin gene (). Previously, we applied CRISPR-Cas9-mediated "single-cut" genome editing to correct diverse genetic mutations in...
4.
Amoasii L, Li H, Zhang Y, Min Y, Sanchez-Ortiz E, Shelton J, et al.
Nat Commun
. 2019 Oct;
10(1):4537.
PMID: 31586095
Duchenne muscular dystrophy (DMD) is a fatal genetic disorder caused by mutations in the dystrophin gene. To enable the non-invasive analysis of DMD gene correction strategies in vivo, we introduced...
5.
Min Y, Li H, Rodriguez-Caycedo C, Mireault A, Huang J, Shelton J, et al.
Sci Adv
. 2019 Mar;
5(3):eaav4324.
PMID: 30854433
Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), which is characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete exon 44 of the dystrophin gene...
6.
Amoasii L, Long C, Li H, Mireault A, Shelton J, Sanchez-Ortiz E, et al.
Sci Transl Med
. 2017 Dec;
9(418).
PMID: 29187645
Duchenne muscular dystrophy (DMD) is a severe, progressive muscle disease caused by mutations in the dystrophin gene. The majority of DMD mutations are deletions that prematurely terminate the dystrophin protein....
7.
Long C, Amoasii L, Mireault A, McAnally J, Li H, Sanchez-Ortiz E, et al.
Science
. 2016 Jan;
351(6271):400-3.
PMID: 26721683
CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene. To correct DMD by skipping...
8.
Long C, McAnally J, Shelton J, Mireault A, Bassel-Duby R, Olson E
Science
. 2014 Aug;
345(6201):1184-1188.
PMID: 25123483
Duchenne muscular dystrophy (DMD) is an inherited X-linked disease caused by mutations in the gene encoding dystrophin, a protein required for muscle fiber integrity. DMD is characterized by progressive muscle...