Below are the most recent publications written about "Dystrophin" by people in Profiles.
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Hern?ndez Rodr?guez MY, Biswas DD, Slyne AD, Lee J, Scarrow E, Abdelbarr SM, Daniels H, O'Halloran KD, Ferreira LF, Gersbach CA, ElMallah MK. Respiratory pathology in the mdx/utrn -/- mouse: A murine model for Duchenne Muscular Dystrophy (DMD). PLoS One. 2025; 20(2):e0316295.
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Susorov D, Echeverria D, Khvorova A, Korostelev AA. mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs). Nucleic Acids Res. 2024 Aug 27; 52(15):8687-8701.
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Roger AL, Biswas DD, Huston ML, Le D, Bailey AM, Pucci LA, Shi Y, Robinson-Hamm J, Gersbach CA, ElMallah MK. Respiratory characterization of a humanized Duchenne muscular dystrophy mouse model. Respir Physiol Neurobiol. 2024 08; 326:104282.
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Hart CC, Lee YI, Xie J, Gao G, Lin BL, Hammers DW, Sweeney HL. Potential limitations of microdystrophin gene therapy for Duchenne muscular dystrophy. JCI Insight. 2024 May 07; 9(11).
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Sarker S, Eshaque TB, Soorajkumar A, Nassir N, Zehra B, Kanta SI, Rahaman MA, Islam A, Akter S, Ali MK, Mim RA, Uddin KMF, Chowdhury MSJ, Shams N, Baqui MA, Lim ET, Akter H, Woodbury-Smith M, Uddin M. Mutational spectrum and phenotypic variability of Duchenne muscular dystrophy and related disorders in a Bangladeshi population. Sci Rep. 2023 12 06; 13(1):21547.
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Engelbeen S, O'Reilly D, Van De Vijver D, Verhaart I, van Putten M, Hariharan V, Hassler M, Khvorova A, Damha MJ, Aartsma-Rus A. Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy. Nucleic Acid Ther. 2023 12; 33(6):348-360.
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Lek A, Wong B, Keeler A, Blackwood M, Ma K, Huang S, Sylvia K, Batista AR, Artinian R, Kokoski D, Parajuli S, Putra J, Carreon CK, Lidov H, Woodman K, Pajusalu S, Spinazzola JM, Gallagher T, LaRovere J, Balderson D, Black L, Sutton K, Horgan R, Lek M, Flotte T. Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. N Engl J Med. 2023 Sep 28; 389(13):1203-1210.
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D'Ambrosio ES, Mendell JR. Evolving Therapeutic Options for the Treatment of Duchenne Muscular Dystrophy. Neurotherapeutics. 2023 Oct; 20(6):1669-1681.
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Mhandire DZ, Burns DP, Roger AL, O'Halloran KD, ElMallah MK. Breathing in Duchenne muscular dystrophy: translation to therapy. J Physiol. 2022 08; 600(15):3465-3482.
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Flotte TR. Precision of Exon Skipping with U7 Constructs. Hum Gene Ther. 2021 11; 32(21-22):1315-1316.