268 Virus-like particle–mediated Cas9/gRNA delivery with a CFTR super exon rescues CFTR function in differentiated primary human bronchial epithelial cells
Aspiras, A., Bell, A., Misiolek, R. Valdez, Yan, K., Macadino, C., Bukis, K., Coote, K., Mense, M., Cotton, C., Valley, H.
Published in Journal of cystic fibrosis (01.09.2024)
Published in Journal of cystic fibrosis (01.09.2024)
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Journal Article
257 Evaluating genetic components to move toward a highly effective CFTR super exon
Yan, K., Bell, A., Macadino, C., Conte, J., Armstrong, M., Cheng, Y., Coote, K., Allaire, N., Sivachenko, A., Mense, M., Cotton, C., Valley, H.
Published in Journal of cystic fibrosis (01.10.2023)
Published in Journal of cystic fibrosis (01.10.2023)
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Journal Article
261 Optimized CFTR E2-27 super exon cargos rescue CFTR function
Yan, K., Macadino, C., Bukis, K., Bell, A., Chado, M., Armstrong, M., Conte, J., Cheng, Y., Tabak, B., Coote, K., Allaire, N., Sivachenko, A., Mense, M., Cotton, C., Valley, H.
Published in Journal of cystic fibrosis (01.09.2024)
Published in Journal of cystic fibrosis (01.09.2024)
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Journal Article
663 Modulation of cystic fibrosis transmembrane conductance regulator intron 22 alternative polyadenylation use may have therapeutic potential for the treatment of certain 3′ cystic fibrosis transmembrane conductance regulator premature termination codon variants
Allaire, N., Yoon, J., Armstrong, M., Valley, H., Macadino, C., Sivachenko, A., Conte, J., Tabak, B., Bihler, H., Cheng, Y., Coote, K., Cotton, C., Mense, M.
Published in Journal of cystic fibrosis (01.10.2022)
Published in Journal of cystic fibrosis (01.10.2022)
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