Carlon, Marianne S.

Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis

Alton, E. W. F. W., Beekman, J. M., Boyd, A. C., Brand, J., Carlon, M. S., Connolly, M. M., Chan, M., Conlon, S., Davidson, H. E., Davies, J. C., et al. (2017) · Thorax

Virus-based gene therapy researchCystic Fibrosis Research Advances

Cited by 150

Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing

Maule, G., Casini, A., Montagna, C., Ramalho, A. S., De Boeck, K., Debyser, Z., Carlon, M. S., Petris, G., & Cereseto, A. (2019) · Nature Communications

CRISPR and Genetic EngineeringCystic Fibrosis Research Advances

Cited by 86

rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice

Vidović, D., Carlon, M. S., da Cunha, M. F., Dekkers, J. F., Hollenhorst, M. I., Bijvelds, M. J. C., Ramalho, A. S., Van den Haute, C., Ferrante, M., Baekelandt, V., et al. (2016) · American Journal of Respiratory and Critical Care Medicine

Cystic Fibrosis Research AdvancesVirus-based gene therapy research

Cited by 74

Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells

Bulcaen, M., Kortleven, P., Liu, R. B., Maule, G., Dreano, E., Kelly, M., Ensinck, M. M., Thierie, S., Smits, M., Ciciani, M., et al. (2024) · Cell Reports Medicine

Cystic Fibrosis Research AdvancesCRISPR and Genetic Engineering

Cited by 69

Unagi: Deep Generative Model for Deciphering Cellular Dynamics and In-Silico Drug Discovery in Complex Diseases

Zheng, Y., Schupp, J., Adams, T., Clair, G., Justet, A., Ahangari, F., Yan, X., Hansen, P., Carlon, M., Cortesi, E., et al. (2023) · Nature Biomedical Engineering

Single-cell and spatial transcriptomicsGene Regulatory Network Analysis

Cited by 34

Nanoblades allow high-level genome editing in murine and human organoids

Tiroille, V., Krug, A., Bokobza, E., Kahi, M., Bulcaen, M., Ensinck, M. M., Geurts, M. H., Hendriks, D., Vermeulen, F., Larbret, F., et al. (2023) · Molecular Therapy - Nucleic Acids

CRISPR and Genetic EngineeringRNA Interference and Gene Delivery

Cited by 25

Immunological Ignorance Allows Long-Term Gene Expression After Perinatal Recombinant Adeno-Associated Virus-Mediated Gene Transfer to Murine Airways

Carlon, M. S., Vidović, D., Dooley, J., da Cunha, M. M., Maris, M., Lampi, Y., Toelen, J., Van den Haute, C., Baekelandt, V., Deprest, J., et al. (2014) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 19