ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia by Morini, Elisabetta, Gao, Dadi, Montgomery, Connor M., Salani, Monica, Mazzasette, Chiara, Krussig, Tobias A., Swain, Brooke, Dietrich, Paula, Narasimhan, Jana, Gabbeta, Vijayalakshmi, Dakka, Amal, Hedrick, Jean, Zhao, Xin, Weetall, Marla, Naryshkin, Nikolai A., Wojtkiewicz, Gregory G., Ko, Chien-Ping, Talkowski, Michael E., Dragatsis, Ioannis, Slaugenhaupt, Susan A.

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Astrocytes influence the severity of spinal muscular atrophy by Rindt, Hansjörg, Feng, Zhihua, Mazzasette, Chiara, Glascock, Jacqueline J, Valdivia, David, Pyles, Noah, Crawford, Thomas O, Swoboda, Kathryn J, Patitucci, Teresa N, Ebert, Allison D, Sumner, Charlotte J, Ko, Chien-Ping, Lorson, Christian L

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Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy by Osman, Erkan Y, Washington, Charles W, Kaifer, Kevin A, Mazzasette, Chiara, Patitucci, Teresa N, Florea, Kyra M, Simon, Madeline E, Ko, Chien-Ping, Ebert, Allison D, Lorson, Christian L

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Extrasynaptic muscarinic acetylcholine receptors on neuronal cell bodies regulate presynaptic function in Caenorhabditis elegans by Chan, Jason P, Staab, Trisha A, Wang, Han, Mazzasette, Chiara, Butte, Zara, Sieburth, Derek

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Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy by Cobb, Melissa S, Rose, Ferril F, Rindt, Hansjörg, Glascock, Jacqueline J, Shababi, Monir, Miller, Madeline R, Osman, Erkan Y, Yen, Pei-Fen, Garcia, Michael L, Martin, Brittanie R, Wetz, Mary J, Mazzasette, Chiara, Feng, Zhihua, Ko, Chien-Ping, Lorson, Christian L

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