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Allele-specific silencing therapy for Dynamin 2-related dominant centronuclear myopathy.
EMBO Mol Med. 2018 Feb;10(2):239-253. doi: 10.15252/emmm.201707988.
EMBO Mol Med. 2018.
PMID: 29246969
Free PMC article.
Allele-Specific CRISPR/Cas9 Correction of a Heterozygous DNM2 Mutation Rescues Centronuclear Myopathy Cell Phenotypes.
Rabai A, Reisser L, Reina-San-Martin B, Mamchaoui K, Cowling BS, Nicot AS, Laporte J.
Rabai A, et al.
Mol Ther Nucleic Acids. 2019 Jun 7;16:246-256. doi: 10.1016/j.omtn.2019.02.019. Epub 2019 Feb 27.
Mol Ther Nucleic Acids. 2019.
PMID: 30925452
Free PMC article.
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Amphiphysin (BIN1) negatively regulates dynamin 2 for normal muscle maturation.
Cowling BS, Prokic I, Tasfaout H, Rabai A, Humbert F, Rinaldi B, Nicot AS, Kretz C, Friant S, Roux A, Laporte J.
Cowling BS, et al. Among authors: rabai a.
J Clin Invest. 2017 Dec 1;127(12):4477-4487. doi: 10.1172/JCI90542. Epub 2017 Nov 13.
J Clin Invest. 2017.
PMID: 29130937
Free PMC article.
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Clinical and genetic aspect of 30 tunisian families with febrile seizures.
Kamoun Feki F, Fendri Kriaa N, Kolsi D, Rabai A, Fakhfakh F, Charfi Triki C.
Kamoun Feki F, et al. Among authors: rabai a.
Tunis Med. 2019 Apr;97(4):525-532.
Tunis Med. 2019.
PMID: 31729702
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