Below are the most recent publications written about "Exons" by people in Profiles.
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Blitek M, Le Coz O, Ogor V, Fakih HH, Khvorova A, Goyenvalle A. Efficient Downregulation of Flt-1 Mediated by Splice Switching ASO in Murine Endothelial Cells. Nucleic Acid Ther. 2026 Jun; 36(3):147-157.
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Lee A, Gentzler A, Fitzpatrick D, Raju Ponny S, Aydemir O, Han MS, Lewis CA, Gao G, Davis RJ. Mutually exclusive alternative pre-mRNA splicing promotes adaptive metabolic stress signaling by JNK. Proc Natl Acad Sci U S A. 2026 Mar 24; 123(12):e2527162123.
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Groenwold ET, Montulet A, Stan T, van de Vijver D, McCorquodale D, McHugh N, Echeverria D, Winter CT, van de Wal M, O'Reilly D, Khvorova A, Aartsma-Rus A, Damha MJ. Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study. Nucleic Acid Ther. 2026 Apr; 36(2):100-108.
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Calhoun CCS, Capps MES, Muya K, Gannaway WC, Martina V, Conklin CL, Klein MC, Webster JM, Torija-Olson EG, Thyme SB. Removal of developmentally regulated microexons has a minimal impact on larval zebrafish brain morphology and function. Elife. 2025 Nov 18; 13.
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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 11 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 10; 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.