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Paper

Frederiksen, Sabrina B, Holm, Lise L, Larsen, Martin R, Doktor, Thomas K, Andersen, Henriette S, Hastings, Michelle L, Hua, Yimin, Krainer, Adrian R, Andresen, Brage S (March 2021) Identification of SRSF10 as a regulator of SMN2 ISS-N1. Human Mutation, 42 (3). pp. 246-260. ISSN 1059-7794

Sheng, L., Rigo, F., Bennett, C. F., Krainer, A. R., Hua, Y. (April 2020) Comparison of the Efficacy of MOE and PMO Modifications of Systemic Antisense Oligonucleotides in a Severe SMA Mouse Model. Nucleic Acids Research, 6 (48). pp. 2853-2865. ISSN 0305-1048 (Public Dataset)

Sinha, R., Kim, Y. J., Nomakuchi, T., Sahashi, K., Hua, Y., Rigo, F., Bennett, C. F., Krainer, A. R. (June 2018) Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice. Nucleic Acids Res, 46 (10). pp. 4833-4844. ISSN 0305-1048

Doktor, T. K., Hua, Y., Andersen, H. S., Broner, S., Liu, Y. H., Wieckowska, A., Dembic, M., Bruun, G. H., Krainer, A. R., Andresen, B. S. (January 2017) RNA-sequencing of a mouse-model of spinal muscular atrophy reveals tissue-wide changes in splicing of U12-dependent introns. Nucleic Acids Res, 45 (1). pp. 395-416. ISSN 1362-4962 (Electronic)0305-1048 (Linking)

Anczukow, O., Akerman, M., Clery, A., Wu, J., Shen, C., Shirole, N. H., Raimer, A., Sun, S., Jensen, M. A., Hua, Y., Allain, F. H., Krainer, A. R. (October 2015) SRSF1-Regulated Alternative Splicing in Breast Cancer. Mol Cell, 60 (1). pp. 105-17. ISSN 1097-4164 (Electronic)1097-2765 (Linking)

Staropoli, John F., Li, Huo, Chun, Seung J., Allaire, Norm, Cullen, Patrick, Thai, Alice, Fleet, Christina M., Hua, Yimin, Bennett, C. Frank, Krainer, Adrian R., Kerr, Doug, McCampbell, Alexander, Rigo, Frank, Carulli, John P. (January 2015) Rescue of gene-expression changes in an induced mouse model of spinal muscular atrophy by an antisense oligonucleotide that promotes inclusion of SMN2 exon 7. Genomics, 105 (4). pp. 220-228. ISSN 0888-7543

Xiong, H. Y., Alipanahi, B., Lee, L. J., Bretschneider, H., Merico, D., Yuen, R. K., Hua, Y., Gueroussov, S., Najafabadi, H. S., Hughes, T. R., Morris, Q., Barash, Y., Krainer, A. R., Jojic, N., Scherer, S. W., Blencowe, B. J., Frey, B. J. (December 2014) RNA splicing. The human splicing code reveals new insights into the genetic determinants of disease. Science, 347 (6218). ISSN 0036-8075

Rigo, F., Chun, S. J., Norris, D. A., Hung, G., Lee, S., Matson, J., Fey, R. A., Gaus, H., Hua, Y., Grundy, J. S., Krainer, A. R., Henry, S. P., Bennett, C. F. (July 2014) Pharmacology of a central nervous system delivered 2′-O-methoxyethyl- modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates. Journal of Pharmacology and Experimental Therapeutics, 350 (1). pp. 46-55. ISSN 15210103

Sahashi, K., Ling, K. K., Hua, Y., Wilkinson, J. E., Nomakuchi, T., Rigo, F., Hung, G., Xu, D., Jiang, Y. P., Lin, R. Z., Ko, C. P., Bennett, C. F., Krainer, A. R. (September 2013) Pathological impact of SMN2 mis-splicing in adult SMA mice. EMBO Molecular Medicine, 5 (10). pp. 1586-1601. ISSN 17574676 (ISSN)

Rigo, F., Hua, Y. M., Krainer, A. R., Bennett, C. F. (October 2012) Antisense-based therapy for the treatment of spinal muscular atrophy. Journal of Cell Biology, 199 (1). pp. 21-25. ISSN 0021-9525

Sahashi, K., Hua, Y., Ling, K. K., Hung, G., Rigo, F., Horev, G., Katsuno, M., Sobue, G., Ko, C. P., Bennett, C. F., Krainer, A. R. (August 2012) TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animals. Genes Dev, 26 (16). pp. 1874-84. ISSN 1549-5477 (Electronic)0890-9369 (Linking)

Rigo, F., Hua, Y. M., Chun, S. J., Prakash, T. P., Krainer, A. R., Bennett, C. F. (June 2012) Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing. Nature Chemical Biology, 8 (6). pp. 555-561. ISSN 1552-4450

Hua, Y., Krainer, A. R. (2012) Antisense-mediated exon inclusion. Methods Mol Biol, 867. pp. 307-323. ISSN 1940-6029 (Electronic)1064-3745 (Linking)

Hua, Y., Sahashi, K., Rigo, F., Hung, G., Horev, G., Bennett, C. F., Krainer, A. R. (October 2011) Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model. Nature, 478 (7367). pp. 123-6. ISSN 1476-4687 (Electronic) 0028-0836 (Linking)

Rigo, F., Hua, Y. M., Chun, S. J., Krainer, A. R., Bennett, C. F. (October 2011) Modulation of Gene Expression By Oligonucleotide Chemistry-Dependent Recruitment of Proteins to RNA Transcripts. Nucleic Acid Therapeutics, 21 (5). A53-A54. ISSN 2159-3337

Krainer, A. R., Hua, Y. M., Sahashi, K., Rigo, F., Hung, G. N., Horev, G., Bennett, C. F. (October 2011) Systemic Versus CNS Delivery Of MOE Antisense Oligonucleotide to Correct Defective Splicing in a Severe Mouse Model of Spinal Muscular Atrophy. Nucleic Acid Therapeutics, 21 (5). A57-A57. ISSN 2159-3337

Passini, M. A., Bu, J., Richards, A. M., Kinnecom, C., Sardi, S. P., Stanek, L. M., Hua,  Y., Rigo, F., Matson, J., Hung, G., Kaye, E. M., Shihabuddin, L. S., Krainer, A. R., Bennett, C. F., Cheng, S. H. (March 2011) Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy. Science Translational Medicine, 3 (72). ISSN 19466234 (ISSN)

Hua, Y., Sahashi, K., Hung, G., Rigo, F., Passini, M. A., Bennett, C. F., Krainer, A. R. (August 2010) Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes and Development, 24 (15). pp. 1634-44. ISSN 08909369 (ISSN)

Hua, Y., Vickers, T. A., Okunola, H. L., Bennett, C. F., Krainer, A. R. (April 2008) Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice. Am J Hum Genet, 82 (4). pp. 834-48.

Book Section

Hua, Yimin, Sahashi, Kentaro, Rigo, Frank, Hung, Gene, Bennett, C. Frank, Krainer, AdrianR (2013) Correction of RNA Splicing with Antisense Oligonucleotides as a Therapeutic Strategy for a Neurodegenerative Disease. In: Chembiomolecular Science. Springer Japan, pp. 301-313. ISBN 978-4-431-54037-3

Conference or Workshop Item

Broner, S, Doktor, T, Hua, Y, Larsen, M, Krainer, A, Andresen, B (October 2018) SRSF10 effects splicing of SMN2 exon 7. In: 50th European-Society-of-Human-Genetics (ESHG) Conference.