Search Results - "Ensinck, M"

One Size Does Not Fit All: The Past, Present and Future of Cystic Fibrosis Causal Therapies by Ensinck, Marjolein M., Carlon, Marianne S.

“…Cystic fibrosis (CF) is the most common monogenic disorder, caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Over the last 30…”
Get full text

Novel CFTR modulator combinations maximise rescue of G85E and N1303K in rectal organoids by Ensinck, Marjolein M, De Keersmaecker, Liesbeth, Ramalho, Anabela S, Cuyx, Senne, Van Biervliet, Stephanie, Dupont, Lieven, Christ, Frauke, Debyser, Zeger, Vermeulen, François, Carlon, Marianne S

“…Cystic fibrosis (CF) is a severe monogenic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator ( ) gene. Several types of…”
Get full text

Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor by Amistadi, Simone, Maule, Giulia, Ciciani, Matteo, Ensinck, Marjolein M., De Keersmaecker, Liesbeth, Ramalho, Anabela S., Guidone, Daniela, Buccirossi, Martina, Galietta, Luis J.V., Carlon, Marianne S., Cereseto, Anna

“…Cystic fibrosis (CF) is a genetic disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. The 2789+5G>A CFTR mutation is a quite…”
Get full text

Nanoblades allow high-level genome editing in murine and human organoids by Tiroille, Victor, Krug, Adrien, Bokobza, Emma, Kahi, Michel, Bulcaen, Mattijs, Ensinck, Marjolein M., Geurts, Maarten H., Hendriks, Delilah, Vermeulen, François, Larbret, Frédéric, Gutierrez-Guerrero, Alejandra, Chen, Yu, Van Zundert, Indra, Rocha, Susana, Rios, Anne C., Medaer, Louise, Gijsbers, Rik, Mangeot, Philippe E., Clevers, Hans, Carlon, Marianne S., Bost, Frédéric, Verhoeyen, Els

“…Genome engineering has become more accessible thanks to the CRISPR-Cas9 gene-editing system. However, using this technology in synthetic organs called…”
Get full text

Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells by Bulcaen, Mattijs, Kortleven, Phéline, Liu, Ronald B., Maule, Giulia, Dreano, Elise, Kelly, Mairead, Ensinck, Marjolein M., Thierie, Sam, Smits, Maxime, Ciciani, Matteo, Hatton, Aurelie, Chevalier, Benoit, Ramalho, Anabela S., Casadevall i Solvas, Xavier, Debyser, Zeger, Vermeulen, François, Gijsbers, Rik, Sermet-Gaudelus, Isabelle, Cereseto, Anna, Carlon, Marianne S.

“…Prime editing is a recent, CRISPR-derived genome editing technology capable of introducing precise nucleotide substitutions, insertions, and deletions. Here,…”
Get full text

318 Investigation of CFTR function and epithelial barrier properties at single-cell resolution using multi-electrode array technology by Ensinck, M., Venz, A., Ramalho, A.S., Takalloo, S. Ebrahimi, Vermeulen, F., Braeken, D., Carlon, M.

Get full text

317 Improving N1303K rescue by combining elexacaftor/tezacaftor/ivacaftor with β2 agonist formoterol by Ensinck, M., Ramalho, A.S., Nijs, M., De Keersmaecker, L., Thierie, S., Munck, S., Klaassen, H., Christ, F., Debyser, Z., Vermeulen, F., Carlon, M.

Get full text

P025 Assessment of CFTR modulator combinations in rectal organoids from F508del homozygous patients with cystic fibrosis by Cuyx, S., Ramalho, A.S., Proesmans, M., Boon, M., Ensinck, M., Carlon, M.S., Dupont, L., De Boeck, K., Vermeulen, F.

Get full text

WS16.02 Base editing strategy to repair the CFTR 2789 + 5G > A splicing mutation by Amistadi, S., Maule, G., Ensinck, M., De Keersmaecker, L., Guidone, D., Ramalho, A.S., Vermeulen, F., Carlon, M., Galietta, L.JV, Cereseto, A.

Get full text

P023 Characterisation of CFTR function in a patient with the F508del/ CFTRdup1–3 genotype by Cuyx, S., Ramalho, A., Van Biervliet, S., Proesmans, M., Boon, M., Carlon, M., Ensinck, M., De Boeck, K., Vermeulen, F.

Get full text

271 Prime editing functionally corrects rare and common cystic fibrosis–causing mutations in patient-derived organoids and airway epithelia by Bulcaen, M., Kortleven, P., Liu, R., Maule, G., Dreano, E., Kelly, M., Ensinck, M., Thierie, S., Smits, M., Ciciani, M., Hatton, A., Chevalier, B., Ramalho, A.S., i Solvas, X. Casadevall, Debyser, Z., Vermeulen, F., Gijsbers, R., Sermet-Gaudelus, I., Cereseto, A., Carlon, M.

Get full text

WS09.2 Elexacaftor combinations (partially) restore CFTR function to previously drug-refractory mutations G85E and N1303K by Ensinck, M., De Keersmaecker, L., Ramalho, A.S., Cuyx, S., Van Biervliet, S., De Boeck, K., Christ, F., Debyser, Z., Vermeulen, F., Carlon, M.S.

Get full text

P031 The last 10%: small molecule screening for correctors of rare CFTRprocessing mutations by Ensinck, M., De Keersmaecker, L., Nijs, M., Ramalho, A.S., De Boeck, K., Munck, S., Klaassen, H., Christ, F., Debyser, Z., Carlon, M.S.

Get full text

WS13-1 CFTR processing mutations cause distinct trafficking and functional defects by Ensinck, M., DeKeersmaecker, L., Heylen, L., Ramalho, A.S., De Boeck, K., Christ, F., Debyser, Z., Carlon, M.

Get full text

WS11.3 The last 10%: small molecule screening for correctors of rare CFTR processing mutations by Ensinck, M., De Keersmaecker, L., Nijs, M., Ramalho, A.S., De Boeck, K., Munck, S., Klaassen, H., Christ, F., Debyser, Z., Carlon, M.S.

Get full text

A31 MECHANISMS IN CYSTIC FIBROSIS AND OTHER BRONCHIECTATIC DISEASES: Development Of Recombinant Adeno-Associated Viral Vector (raav) Gene Therapy For Cystic Fibrosis by Carlon, M S, Vidovic, D, Ensinck, M, Da Cunha, M F, Dekkers, J F, Bijvelds, M, De Jonge, H, Ramalho, A S, De Boeck, K, Sermet-Gaudelus, I, Gijsbers, R, Edelman, A, Beekman, J, Debyser, Z

“…[...]we administered rAAV2/5-CFTRÄR to mouse airways via nasal instillation and assessed a potential functional rescue by in vivo nasal potential differences…”
Get full text

P-26EEFECT OF AGE OF DRINKING ONSET ON PATTERNS OF ALCOHOL, TOBACCO, MARIHUANA, STIMULANTS AND HALLUCINOGENS USE, DURING YOUNG ADULTHOOD by Pilatti, A., Fernández Calderón, F., Bonino, P., Ensinck Atienza, M. G., Rivarola Montejano, G., Pautassi, R. M.

Get full text