Increased polyamines as protective disease modifiers in congenital muscular dystrophy von Kemaladewi, D U, Benjamin, J S, Hyatt, E, Ivakine, E A, Cohn, R D

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CRISPR/Cas9-mediated exon inclusion in Lama2 gene alleviates dystrophic pathology in MDC1A mouse model von Kemaladewi, D, Hyatt, E, Ivakine, Z, Cohn, R

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G.O.6 von Yoon, G, Malam, Z, Paton, T, Marshall, C, Hyatt, E, Ivakine, Z, Kemaladewi, D, Forge, C, Lee, K.S, Hawkins, C, Cohn, R.D

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P.347 - CRISPR/Cas9-mediated exon inclusion in Lama2 gene alleviates dystrophic pathology in MDC1A mouse model von Kemaladewi, D., Hyatt, E., Ivakine, Z., Cohn, R.

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NEW THERAPEUTIC APPROACHES AND THEIR READOUT von Kemaladewi, D., Bassi, P., Lindsay, K., Erwood, S., Hyatt, E., Place, K., Marks, R., Gawlik, K., Durbeej, M., Ivakine, E., Cohn, R.

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NEW THERAPEUTIC APPROACHES AND THEIR READOUT: O.14A mutation-independent approach via transcriptional upregulation of a disease modifier gene rescues muscular dystrophy in vivo von Kemaladewi, D., Bassi, P., Lindsay, K., Erwood, S., Hyatt, E., Place, K., Marks, R., Gawlik, K., Durbeej, M., Ivakine, E., Cohn, R.

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G.O.6: Lethal disorder of mitochondrial fission caused by mutations in DNM1L von Yoon, G., Malam, Z., Paton, T., Marshall, C., Hyatt, E., Ivakine, Z., Kemaladewi, D., Forge, C., Lee, K.S., Hawkins, C., Cohn, R.D.

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O.13 Interference of myostatin and TGF-beta signaling by antisense-mediated exon skipping in ALK4/5 receptors von Kemaladewi, D.U, van Heiningen, S.H, Aartsma-Rus, A, van Ommen, G.J.B, ten Dijke, P, t Hoen, P.A.C, Hoogaars, W.M.H

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O.10 Effect of combined treatment with soluble activin receptor IIB and AAV-U7-mediated dystrophin exon skipping on muscle function in mdx mice von Hoogaars, W.M.H, Mouisel, E, Relizani, K, Pasternack, A, Hourde, C, Precigout, G, Ferry, A, Garcia, L, Kemaladewi, D.U, Aartsma-Rus, A, Ritvos, O, t Hoen, P.A.C, Amthor, H

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Expanding the Boundaries of RNA Sequencing as a Diagnostic Tool for Rare Mendelian Disease von Gonorazky, Hernan D., Naumenko, Sergey, Ramani, Arun K., Nelakuditi, Viswateja, Mashouri, Pouria, Wang, Peiqui, Kao, Dennis, Ohri, Krish, Viththiyapaskaran, Senthuri, Tarnopolsky, Mark A., Mathews, Katherine D., Moore, Steven A., Osorio, Andres N., Villanova, David, Kemaladewi, Dwi U., Cohn, Ronald D., Brudno, Michael, Dowling, James J.

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Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders von Wojtal, Daria, Kemaladewi, Dwi U., Malam, Zeenat, Abdullah, Sarah, Wong, Tatianna W.Y., Hyatt, Elzbieta, Baghestani, Zahra, Pereira, Sergio, Stavropoulos, James, Mouly, Vincent, Mamchaoui, Kamel, Muntoni, Francesco, Voit, Thomas, Gonorazky, Hernan D., Dowling, James J., Wilson, Michael D., Mendoza-Londono, Roberto, Ivakine, Evgueni A., Cohn, Ronald D.

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A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene von Kemaladewi, Dwi U., Bassi, Prabhpreet S., Erwood, Steven, Al-Basha, Dhekra, Gawlik, Kinga I., Lindsay, Kyle, Hyatt, Elzbieta, Kember, Rebekah, Place, Kara M., Marks, Ryan M., Durbeej, Madeleine, Prescott, Steven A., Ivakine, Evgueni A., Cohn, Ronald D.

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Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism von Kemaladewi, Dwi U, Maino, Eleonora, Hyatt, Elzbieta, Hou, Huayun, Ding, Maylynn, Place, Kara M, Zhu, Xinyi, Bassi, Prabhpreet, Baghestani, Zahra, Deshwar, Amit G, Merico, Daniele, Xiong, Hui Y, Frey, Brendan J, Wilson, Michael D, Ivakine, Evgueni A, Cohn, Ronald D

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Expanding the Boundaries of RNA Sequencing as a Diagnostic Tool for Rare Mendelian Disease von Gonorazky, Hernan D., Naumenko, Sergey, Ramani, Arun K., Nelakuditi, Viswateja, Mashouri, Pouria, Wang, Peiqui, Kao, Dennis, Ohri, Krish, Viththiyapaskaran, Senthuri, Tarnopolsky, Mark A., Mathews, Katherine D., Moore, Steven A., Osorio, Andres N., Villanova, David, Kemaladewi, Dwi U., Cohn, Ronald D., Brudno, Michael, Dowling, James J.

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Exon Snipping in Duchenne Muscular Dystrophy von Kemaladewi, Dwi U, Cohn, Ronald D

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Development of therapeutic genome engineering in laminin-α2-deficient congenital muscular dystrophy von Kemaladewi, Dwi U., Cohn, Ronald D.

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559. Spell Checking Nature: A Therapeutic Use of the CRISPR/Cas9 System in Duchenne Muscular Dystrophy von Wojtal, Daria, Kemaladewi, Dwi U., Malam, Zeenat, Abdullah, Sarah, Hyatt, Elzbieta, Huang, Victoria, Mouly, Vincent, Voit, Thomas, Muntoni, Francesco, Ivakine, Evgueni A., Cohn, Ronald D.

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GENE EDITING SYSTEM FOR CORRECTING SPLICING DEFECTS von KEMALADEWI, Dwi Utami, IVAKINE, Evgueni, COHN, Ronald D

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GENE EDITING SYSTEM FOR CORRECTING SPLICING DEFECTS von Cohn, Ronald D, Ivakine, Evgueni, Kemaladewi, Dwi Utami

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GENE EDITING SYSTEM FOR CORRECTING SPLICING DEFECTS von KEMALADEWI, Dwi Utami, IVAKINE, Evgueni, COHN, Ronald D

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