Below are the most recent publications written about "Dystrophin" by people in Profiles.
-
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.
-
Flotte TR. Precision of Exon Skipping with U7 Constructs. Hum Gene Ther. 2021 11; 32(21-22):1315-1316.
-
Pan X, Yue Y, Boftsi M, Wasala LP, Tran NT, Zhang K, Pintel DJ, Tai PWL, Duan D. Rational engineering of a functional CpG-free ITR for AAV gene therapy. Gene Ther. 2022 06; 29(6):333-345.
-
Dhindsa JS, McCall AL, Strickland LM, Fusco AF, Kahn AF, ElMallah MK. Motor axonopathies in a mouse model of Duchenne muscular dystrophy. Sci Rep. 2020 06 02; 10(1):8967.
-
Aartsma-Rus A, Krieg AM. FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga. Nucleic Acid Ther. 2017 02; 27(1):1-3.
-
Toh ZY, Thandar Aung-Htut M, Pinniger G, Adams AM, Krishnaswarmy S, Wong BL, Fletcher S, Wilton SD. Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies. PLoS One. 2016; 11(1):e0145620.
-
Vieira NM, Elvers I, Alexander MS, Moreira YB, Eran A, Gomes JP, Marshall JL, Karlsson EK, Verjovski-Almeida S, Lindblad-Toh K, Kunkel LM, Zatz M. Jagged 1 Rescues the Duchenne Muscular Dystrophy Phenotype. Cell. 2015 Nov 19; 163(5):1204-1213.
-
Kole R, Krieg AM. Exon skipping therapy for Duchenne muscular dystrophy. Adv Drug Deliv Rev. 2015 Jun 29; 87:104-7.
-
Spinazzola JM, Smith TC, Liu M, Luna EJ, Barton ER. Gamma-sarcoglycan is required for the response of archvillin to mechanical stimulation in skeletal muscle. Hum Mol Genet. 2015 May 01; 24(9):2470-81.
-
Tandon A, Jefferies JL, Villa CR, Hor KN, Wong BL, Ware SM, Gao Z, Towbin JA, Mazur W, Fleck RJ, Sticka JJ, Benson DW, Taylor MD. Dystrophin genotype-cardiac phenotype correlations in Duchenne and Becker muscular dystrophies using cardiac magnetic resonance imaging. Am J Cardiol. 2015 Apr 01; 115(7):967-71.