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2012, Methods in molecular biology, ISBN 9781617797668, Volume 867., xi, 440
Book
2012, Methods in molecular biology, ISBN 9781617797675, Volume 867
Web Resource
Ophthalmology, ISSN 0161-6420, 2016, Volume 123, Issue 6, pp. 1375 - 1385
Journal Article
Cell, ISSN 0092-8674, 11/2016, Volume 167, Issue 5, pp. 1144 - 1144
Exondys 51 is the first therapy for Duchenne muscular dystrophy (DMD) to have been granted accelerated approval by the FDA. Approval was granted based on using... 
BIOCHEMISTRY & MOLECULAR BIOLOGY | CELL BIOLOGY | Genetic Therapy | Dystrophin - genetics | Exons | United States | Humans | Reading Frames | Muscular Dystrophy, Duchenne - genetics | Muscular Dystrophy, Duchenne - therapy | United States Food and Drug Administration | Drug approval | Dystrophin | Utrophin | Stem cells | Index Medicus
Journal Article
Journal Article
Gene Therapy, ISSN 0969-7128, 01/2014, Volume 21, Issue 1, pp. 52 - 59
Journal Article
The Oncologist, ISSN 1083-7159, 04/2016, Volume 21, Issue 4, pp. 481 - 486
摘要 背景 . 非小细胞肺癌 (NSCLC) 包含的特异性遗传改变可能对靶向疗法高度敏感。 材料与方法 . 我们对 54 例不同疾病分期且从不吸烟、无驱动突变的 NSCLC 患者进行了靶基因重排检测。由于 MET 外显子 14 跳读是最常见的改变, 因此我们当前的分子谱板纳入了这一改变。本研究特别调查了麻省总院... 
Targeted therapy | MET | Lung cancer | exon 14 skipping | MET exon 14 skipping | GEFITINIB | RESPONSIVENESS | ACTIVATION | SENSITIVITY | TUMORS | INHIBITION | GENE | ONCOLOGY | CRIZOTINIB | MUTATIONS | Carcinoma, Non-Small-Cell Lung - pathology | Smoking - adverse effects | Carcinoma, Non-Small-Cell Lung - genetics | Proto-Oncogene Proteins c-met - antagonists & inhibitors | Humans | Middle Aged | Exons - genetics | Male | Molecular Targeted Therapy | Proto-Oncogene Proteins c-met - genetics | Neoplasm Recurrence, Local - pathology | Carcinoma, Non-Small-Cell Lung - therapy | Aged, 80 and over | Female | Neoplasm Recurrence, Local - genetics | Aged | Gene Rearrangement - genetics | Neoplasm Staging | Index Medicus | 14 | Lung Cancer | 29
Journal Article
Nature Medicine, ISSN 1078-8956, 2015, Volume 21, Issue 3, pp. 270 - 275
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 01/2016, Volume 126, Issue 1, pp. 68 - 84
Journal Article
Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 12/2017, Volume 9, Issue C, pp. 155 - 161
Locked nucleic acid is a prominent nucleic acid analog with unprecedented target binding affinity to cDNA and RNA oligonucleotides and shows remarkable... 
locked nucleic acid | nucleic acid chemistry | antisense oligonucleotide | Duchenne muscular dystrophy | exon-skipping | modified nucleotides | chemically-modified nucleotides | MDX | THERAPEUTICS | MEDICINE, RESEARCH & EXPERIMENTAL | POTENT | THERAPY | RNA | ANALOGS | STABILITY | MOUSE | DUCHENNE MUSCULAR-DYSTROPHY | ETEPLIRSEN
Journal Article
Annals of Neurology, ISSN 0364-5134, 06/2009, Volume 65, Issue 6, pp. 667 - 676
Journal Article