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Orphanet Journal of Rare Diseases, ISSN 1750-1172, 2015, Volume 10, Issue Suppl 2, p. O34
Journal Article
by Chatrchyan, S and Khachatryan, V and Sirunyan, A.M and Tumasyan, A and Adam, N and Adam, W and Aguilo, E and Bergauer, T and Dragicevic, M and Erö, J and Fabjan, C and Friedl, M and Frühwirth, R and Ghete, V.M and Hammer, J and Hoch, M and Hörmann, N and Hrubec, J and Jeitler, M and Kiesenhofer, W and Knünz, V and Krammer, M and Krätschmer, I and Liko, D and Majerotto, W and Mikulec, I and Pernicka, M and Rahbaran, B and Rohringer, H and Rohringer, C and Schöfbeck, R and Strauss, J and Szoncsó, F and Taurok, A and Waltenberger, W and Walzel, G and Widl, E and Wulz, C.-E and Chekhovsky, V and Emeliantchik, I and Litomin, A and Makarenko, V and Mossolov, V and Shumeiko, N and Solin, A and Stefanovitch, R and Suarez Gonzalez, J and Fedorov, A and Korzhik, M and Missevitch, O and Zuyeuski, R and Bansal, M and Bansal, S and Beaumont, W and Cornelis, T and De Wolf, E.A and Druzhkin, D and Janssen, X and Luyckx, S and Mucibello, L and Ochesanu, S and Roland, B and Roland, C and Roland, G and Rougny, R and Selvaggi, G and Selvaggi, M and Staykova, Z and Van Haevermaet, H and Van Mechelen, P and Van Remortel, N and Van Spilbeeck, A and Blekman, F and Blyweert, S and DʼHondt, J and Devroede, O and Gonzalez Suarez, R and Goorens, R and Kalogeropoulos, A and Maes, M and Olbrechts, A and Tavernier, S and Van Doninck, W and Van Lancker, L and Van Mulders, P and Van Onsem, G.P and Villella, I and Clerbaux, B and De Lentdecker, G and Dero, V and Dewulf, J.P and Gay, A.P.R and Hreus, T and Léonard, A and Marage, P.E and Mohammadi, A and Reis, T and Rugovac, S and Thomas, S and Thomas, M and ... and CMS Collaboration and Los Alamos National Lab. (LANL), Los Alamos, NM (United States) and Argonne National Lab. (ANL), Argonne, IL (United States) and Fermi National Accelerator Lab. (FNAL), Batavia, IL (United States) and Lawrence Livermore National Lab. (LLNL), Livermore, CA (United States)
Physics Letters B, ISSN 0370-2693, 09/2012, Volume 716, Issue 1, pp. 30 - 61
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2013, Volume 21, Issue 5, pp. 920 - 921
Approximately 1 in 1,000 babies is born with hereditary loss of hearing, translating into 40,000 newborns affected by deafness in the United States alone. The... 
MEDICINE, RESEARCH & EXPERIMENTAL | RNA | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | GENETICS & HEREDITY | MUSCULAR-DYSTROPHY | RESTORATION | ANTISENSE OLIGONUCLEOTIDES | Carrier Proteins - genetics | Usher Syndromes - therapy | Animals | Usher Syndromes - genetics | Humans | Oligonucleotides, Antisense - therapeutic use | Index Medicus
Journal Article
Lancet, The, ISSN 0140-6736, 2011, Volume 378, Issue 9791, pp. 595 - 605
Journal Article
Scientific reports, ISSN 2045-2322, 05/2019, Volume 9, Issue 1, pp. 7672 - 8
A cytosine to thymine mutation at nucleotide 654 of human β-globin intron 2 (β ) is one of the most common mutations causing β-thalassaemia in Chinese and... 
Transcription | Splicing | Oligonucleotides | Thymine | Thalassemia | Protein biosynthesis | mRNA | Erythroid cells | snRNA | Progenitor cells | Stem cells | Hemoglobin | Cytosine | Mutation
Journal Article
Lancet Neurology, The, ISSN 1474-4422, 2009, Volume 8, Issue 10, pp. 918 - 928
Journal Article
Journal Article
PLoS ONE, ISSN 1932-6203, 11/2013, Volume 8, Issue 11, pp. e80263 - e80263
Journal Article