Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA von Dooley, Scott J., McDougald, Devin S., Fisher, Krishna J., Bennicelli, Jeanette L., Mitchell, Lloyd G., Bennett, Jean

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Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans -splicing von Walsh, Christopher E, Chao, Hengjun, Mansfield, S Gary, Bartel, Robert C, Hiriyanna, Suja, Mitchell, Lloyd G, Garcia-Blanco, Mariano A

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Development of Polypeptide-based Nanoparticles for Non-viral Delivery of CD22 RNA Trans-splicing Molecule as a New Precision Medicine Candidate Against B-lineage ALL von Uckun, Fatih M., Mitchell, Lloyd G., Qazi, Sanjive, Liu, Yang, Zheng, Nan, Myers, Dorothea E., Song, Ziyuan, Ma, Hong, Cheng, Jianjun

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5′ Trans-Splicing Repair of the PLEC1 Gene von Wally, Verena, Klausegger, Alfred, Koller, Ulrich, Lochmüller, Hanns, Krause, Sabine, Wiche, Gerhard, Mitchell, Lloyd G., Hintner, Helmut, Bauer, Johann W.

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A novel screening system improves genetic correction by internal exon replacement von Koller, Ulrich, Wally, Verena, Mitchell, Lloyd G, Klausegger, Alfred, Murauer, Eva M, Mayr, Elisabeth, Gruber, Christina, Hainzl, Stefan, Hintner, Helmut, Bauer, Johann W

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CD22ΔE12 as a molecular target for corrective repair using RNA trans-splicing: anti-leukemic activity of a rationally designed RNA trans-splicing molecule von Uckun, Fatih M, Qazi, Sanjive, Ma, Hong, Reaman, Gregory H, Mitchell, Lloyd G

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Partial correction of endogenous ΔF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans -splicing von Engelhardt, John F, Liu, Xiaoming, Jiang, Qinshi, Mansfield, S. Gary, Puttaraju, M, Zhang, Yulong, Zhou, Weihong, Cohn, Jonathan A, Garcia-Blanco, Mariano A, Mitchell, Lloyd G

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Spliceosome-mediated RNA trans -splicing as a tool for gene therapy von Garcia-Blanco, Mariano A, Mitchell, Lloyd G, Puttaraju, M, Jamison, Sharon F, Mansfield, S. Gary

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Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fibrosis transmembrane conductance regulator function to polarized human c... von XIAOMING LIU, MEIHUI LUO, ZHANG, Liang N, ZIYING YAN, ZAK, Roman, WEI DING, MANSFIELD, S. Gary, MITCHELL, Lloyd G, ENGELHARDT, John F

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RNA Trans-Splicing Targeting Endogenous β-Globin Pre-Messenger RNA in Human Erythroid Cells von Uchida, Naoya, Washington, Kareem N, Mozer, Brian, Platner, Charlotte, Ballantine, Josiah, Skala, Luke P, Raines, Lydia, Shvygin, Anna, Hsieh, Matthew M, Mitchell, Lloyd G, Tisdale, John F

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5' exon replacement and repair by spliceosome-mediated RNA trans-splicing von Mansfield, S Gary, Clark, Rebecca Hawkins, Puttaraju, M, Kole, Jolanta, Cohn, Jonathan A, Mitchell, Lloyd G, Garcia-Blanco, Mariano A

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Messenger RNA Repair and Restoration of Protein Function by Spliceosome-Mediated RNA Trans-Splicing von Puttaraju, M, DiPasquale, Janet, Baker, Carl C, Mitchell, Lloyd G, Garcia-Blanco, Mariano A

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440. Optimization of RNA Trans-Splicing for the β-Globin Gene; Detecting Trans-Splicing Events Targeting Endogenous β-Globin Pre-mRNA in Human Erythroid Cells von Uchida, Naoya, Platner, Charlotte, Ballantine, Josiah, Hsieh, Matthew M., Mozer, Brian, Mitchell, Lloyd G., Washington, Kareem N., Tisdale, John F.

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CD22ΔE12 as a molecular target for corrective repair using RNA trans-splicing: anti-leukemic activity of a rationally designed RNA trans-splicing moleculeElectronic supplementary i... von Uckun, Fatih M, Qazi, Sanjive, Ma, Hong, Reaman, Gregory H, Mitchell, Lloyd G

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CD22ΔE12 as a molecular target for corrective repair using a RNA trans-splicing strategy: Anti-leukemic activity of a rationally designed RNA trans-splicing molecule von Uckun, Fatih M., Qazi, Sanjive, Ma, Hong, Reaman, Gregory H., Mitchell, Lloyd G.

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Affinity generation of single-stranded DNA for dideoxy sequencing following the polymerase chain reaction von Mitchell, Lloyd G., Merril, Carl R.

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Compositions and methods for correction of heritable ocular disease von Bennett, Jean, Mitchell, Lloyd G, Bennicelli, Jeannette, Dooley, Scott J

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5' Trans-Splicing Repair of the PLEC1 Gene. Commentary von HENGGE, Ulrich R, WALLY, Verena, KLAUSEGGER, Alfred, KOLLER, Ulrich, LOCHMÜLLER, Hanns, KRAUSE, Sabine, WICHE, Gerhard, MITCHELL, Lloyd G, HINTNER, Helmut, BAUER, Johann W

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Compositions and methods for correction of heritable ocular disease von Bennett, Jean, Mitchell, Lloyd G, Bennicelli, Jeannette, Dooley, Scott J

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Compositions and Methods for Correction of Heritable Ocular Disease von Bennett, Jean, Mitchell, Lloyd G, Bennicelli, Jeannette, Dooley, Scott J

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