Below are the most recent publications written about "Exons" by people in Profiles.
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Calvo-Roitberg E, Daniels RF, Pai AA. Challenges in identifying mRNA transcript starts and ends from long-read sequencing data. Genome Res. 2024 Nov 20; 34(11):1719-1734.
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Joos M, Chang TH, Shimamura A, Newburger PE. Absence of Neutropenia in Patients With Early Exon Nonsense Mutations in ELANE : Clinical Evidence to Support Gene Therapy Approaches for Severe Congenital Neutropenia. J Pediatr Hematol Oncol. 2024 08 01; 46(6):e463-e465.
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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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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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Zheng C, Liu B, Dong X, Gaston N, Sontheimer EJ, Xue W. Template-jumping prime editing enables large insertion and exon rewriting in vivo. Nat Commun. 2023 06 08; 14(1):3369.
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Mou H, Eskiocak O, ?zler KA, Gorman M, Yue J, Jin Y, Wang Z, Gao Y, Janowitz T, Meyer HV, Yu T, Wilkinson JE, Kucukural A, Ozata DM, Beyaz S. CRISPR-induced exon skipping of ?-catenin reveals tumorigenic mutants driving distinct subtypes of liver cancer. J Pathol. 2023 04; 259(4):415-427.
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Nicoli ER, Huebecker M, Han ST, Garcia K, Munasinghe J, Lizak M, Latour Y, Yoon R, Glase B, Tyrlik M, Peiravi M, Springer D, Baker EH, Priestman D, Sidhu R, Kell P, Jiang X, Kolstad J, Kuhn AL, Shazeeb MS, Acosta MT, Proia RL, Platt FM, Tifft CJ. Glb1 knockout mouse model shares natural history with type II GM1 gangliosidosis patients. Mol Genet Metab. 2023 02; 138(2):107508.
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Yang X, Triboulet R, Liu Q, Sendinc E, Gregory RI. Exon junction complex shapes the m6A epitranscriptome. Nat Commun. 2022 12 23; 13(1):7904.
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Aartsma-Rus A, Garanto A, van Roon-Mom W, McConnell EM, Suslovitch V, Yan WX, Watts JK, Yu TW. Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides. Nucleic Acid Ther. 2023 01; 33(1):17-25.
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Tolomeo M, Chimienti G, Lanza M, Barbaro R, Nisco A, Latronico T, Leone P, Petrosillo G, Liuzzi GM, Ryder B, Inbar-Feigenberg M, Colella M, Lezza AMS, Olsen RKJ, Barile M. Retrograde response to mitochondrial dysfunctions associated to LOF variations in FLAD1 exon 2: unraveling the importance of RFVT2. Free Radic Res. 2022 Jul-Aug; 56(7-8):511-525.