Search Results - "oligonucleotide"

Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4 by Irene Vázquez-Domínguez, Mert Öktem, Florian A. Winkelaar, Thai Hoang Nguyen, Anita D.M. Hoogendoorn, Eleonora Roschi, Galuh D.N. Astuti, Raoul Timmermans, Nuria Suárez-Herrera, Ilaria Bruno, Albert Ruiz-Llombart, Joseph Brealey, Olivier G. de Jong, Rob W.J. Collin, Enrico Mastrobattista, Alejandro Garanto

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AntimiR targeting of microRNA-134 reduces seizures in a mouse model of Angelman syndrome by Aoife Campbell, Gareth Morris, Albert Sanfeliu, Joana Augusto, Elena Langa, Jaideep C. Kesavan, Ngoc T. Nguyen, Ronan M. Conroy, Jesper Worm, Lukasz Kielpinski, Mads Aaboe Jensen, Meghan T. Miller, Thomas Kremer, Cristina R. Reschke, David C. Henshall

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GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells by Erik van der Wal, Atze J. Bergsma, Tom J.M. van Gestel, Stijn L.M. in 't Groen, Holm Zaehres, Marcos J. Araúzo-Bravo, Hans R. Schöler, Ans T. van der Ploeg, W.W.M. Pim Pijnappel

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Molecular and Genetic Research in Tuberculosis Clinical Practice and Epidemiology by Bahytkul Zhakipbayeva, Sholpan Beisembayeva, Talgat Muminov

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Strategies for In Vivo Screening and Mitigation of Hepatotoxicity Associated with Antisense Drugs by Piotr J. Kamola, Klio Maratou, Paul A. Wilson, Kay Rush, Tanya Mullaney, Tom McKevitt, Paula Evans, Jim Ridings, Probash Chowdhury, Aude Roulois, Ann Fairchild, Sean McCawley, Karen Cartwright, Nigel J. Gooderham, Timothy W. Gant, Kitty Moores, Stephen A. Hughes, Mark R. Edbrooke, Kenneth Clark, Joel D. Parry

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Cell type-specific over-expression of chromosome 21 genes in fibroblasts and fetal hearts with trisomy 21 by Zigman Warren B, Silverman Wayne, Schupf Nicole, Salas Martha, Guo Meirong, Li Chi-Ming, Husain Sameera, Warburton Dorothy, Thaker Harshwardhan, Tycko Benjamin

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ASSOCIATION OF POLYMORPHISM IN BIOTRANSFORMATION SYSTEM GENES CYP1A1 AND GST WITH RISK OF RELAPSE IN CHILDHOOD ACUTE LEUKEMIA by O.A. Gra

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ASSOCIATION OF POLYMORPHISM IN BIOTRANSFORMATION SYSTEM GENES CYP1A1 AND GST WITH RISK OF RELAPSE IN CHILDHOOD ACUTE LEUKEMIA by O.A. Gra

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MYC ASO Impedes Tumorigenesis and Elicits Oncogene Addiction in Autochthonous Transgenic Mouse Models of HCC and RCC by Renumathy Dhanasekaran, Jangho Park, Alekesey Yevtodiyenko, David I. Bellovin, Stacey J. Adam, Anand Rajan KD, Meital Gabay, Hanan Fernando, Julia Arzeno, Vinodhini Arjunan, Sergei Gryanzov, Dean W. Felsher

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Phenotype to genotype characterization by array-comparative genomic hydridization (a-CGH) in case of fetal malformations: A systematic review by Gabriele Tonni, Marcella Palmisano, Ana Cristina Perez Zamarian, Ana Carolina Rabachini Caetano, Eduardo Félix Martins Santana, Alberto Borges Peixoto, Edecio Armbruster-Moraes, Rodrigo Ruano, Edward Araujo Júnior

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Effective CRISPR/Cas9-based nucleotide editing in zebrafish to model human genetic cardiovascular disorders by Federico Tessadori, Helen I. Roessler, Sanne M. C. Savelberg, Sonja Chocron, Sarah M. Kamel, Karen J. Duran, Mieke M. van Haelst, Gijs van Haaften, Jeroen Bakkers

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Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy by Liubov V. Gushchina, Tatyana A. Vetter, Emma C. Frair, Adrienne J. Bradley, Kelly M. Grounds, Jacob W. Lay, Nianyuan Huang, Aisha Suhaiba, Frederick J. Schnell, Gunnar Hanson, Tabatha R. Simmons, Nicolas Wein, Kevin M. Flanigan

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Pharmacokinetics, pharmacodynamics, and safety of fesomersen, a novel antisense inhibitor of factor XI, in healthy Chinese, Japanese, and Caucasian volunteers by Tianxing Liu, Kensei Hashizume, Eva Krieg, Huijun Chen, Yuki Mukaida, Kirstin Thelen, Frauke Friedrichs, Stefan Willmann, Stephan Schwers, Alexander Solms, Rosie Yu

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New cost-effective human leukocyte antigen testing algorithm for screening of human leukocyte antigen-matched related donor in thalassemia major patient pretransplant workup: A sin... by Parvind Singh, Aseem Kumar Tiwari, Vikash Chandra Mishra, Dinesh Chandra, Amit K Bhardwaj, Nikki Dey, Vimarsh Raina

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Effects of a Novel Thiadiazole Derivative with High Anticancer Activity on Cancer Cell Immunogenic Markers: Mismatch Repair System, PD-L1 Expression, and Tumor Mutation Burden by Sofia Sagredou, Panagiotis Dalezis, Eirini Papadopoulou, Maria Voura, Maria V. Deligiorgi, Michail Nikolaou, Mihalis I. Panayiotidis, George Nasioulas, Vasiliki Sarli, Dimitrios T. Trafalis

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Advax4 delta inulin combination adjuvant together with ECMX, a fusion construct of four protective mTB antigens, induces a potent Th1 immune response and protects mice against Myco... by Bruno de Paula Oliveira Santos, Monalisa Martins Trentini, Renato Beilner Machado, Mara Rúbia Nunes Celes, André Kipnis, Nikolai Petrovsky, Ana Paula Junqueira-Kipnis

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HPV detection using primers MY09/MY11 and GP5+/GP6+ in patients with cytologic and/or colposcopic changes by Emanuella Meneses Venceslau, Mauro Muniz Bezerra, Anna Carolina Mota Lopes, Érick Vieira Souza, Alexandre Sherlley Casimiro Onofre, Claudia Moura de Melo, Verônica de Lourdes Sierpe Jeraldo, Fabiana Botelho de Miranda Onofre

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Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression by Irene González-Martínez, Estefanía Cerro-Herreros, Nerea Moreno, Andrea García-Rey, Jorge Espinosa-Espinosa, Marc Carrascosa-Sàez, Diego Piqueras-Losilla, Andrey Arzumanov, David Seoane-Miraz, Yahya Jad, Richard Raz, Matthew J. Wood, Miguel A. Varela, Beatriz Llamusí, Rubén Artero

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Multiplex Reverse Transcription-PCR Assay for Detection of Type A Influenza Virus plus Differentiation of Avian H7 and H9 Hemagglutinin Subtypes in Iran by E Saberfar, A Najafi, Z Goodarzi, H Lashini

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Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy by Estefanía Cerro-Herreros, Irene González-Martínez, Nerea Moreno, Jorge Espinosa-Espinosa, Juan M. Fernández-Costa, Anna Colom-Rodrigo, Sarah J. Overby, David Seoane-Miraz, Javier Poyatos-García, Juan J. Vilchez, Adolfo López de Munain, Miguel A. Varela, Matthew J. Wood, Manuel Pérez-Alonso, Beatriz Llamusí, Rubén Artero

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