[HTML][HTML] One size does not fit all: the past, present and future of cystic fibrosis causal therapies

MM Ensinck, MS Carlon - Cells, 2022 - mdpi.com
Cystic fibrosis (CF) is the most common monogenic disorder, caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene. Over the last 30 years, tremendous …

[HTML][HTML] Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor

S Amistadi, G Maule, M Ciciani, MM Ensinck… - Molecular Therapy, 2023 - cell.com
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 frequent defect …

Novel CFTR modulator combinations maximise rescue of G85E and N1303K in rectal organoids

MM Ensinck, L De Keersmaecker… - ERJ Open …, 2022 - Eur Respiratory Soc
Introduction Cystic fibrosis (CF) is a severe monogenic disorder caused by mutations in the
cystic fibrosis transmembrane conductance regulator (CFTR) gene. Several types of CFTR …

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

…, E Bokobza, M Kahi, M Bulcaen, MM Ensinck… - … Therapy-Nucleic Acids, 2023 - cell.com
Genome engineering has become more accessible thanks to the CRISPR/Cas9 gene editing
system. However, using this technology in synthetic organs called ‘organoids' is still very …

[HTML][HTML] On the corner of models and cure: gene editing in cystic fibrosis

M Ensinck, A Mottais, C Detry, T Leal… - Frontiers in …, 2021 - frontiersin.org
Cystic fibrosis (CF) is a severe genetic disease for which curative treatment is still lacking.
Next generation biotechnologies and more efficient cell-based and in vivo disease models are …

Rewriting CFTR to cure cystic fibrosis

G Maule, M Ensinck, M Bulcaen, MS Carlon - Progress in Molecular Biology …, 2021 - Elsevier
Cystic fibrosis (CF) is an autosomal recessive monogenic disease caused by mutations in
the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene. Although F508del …

[HTML][HTML] Phenotyping of Rare CFTR Mutations Reveals Distinct Trafficking and Functional Defects

M Ensinck, L De Keersmaecker, L Heylen, AS Ramalho… - Cells, 2020 - mdpi.com
Background. The most common CFTR mutation, F508del, presents with multiple cellular
defects. However, the possible multiple defects caused by many rarer CFTR mutations are not …

Transcriptomic analysis of CFTR-impaired endothelial cells reveals a pro-inflammatory phenotype

…, K Brepoels, M Ensinck… - European …, 2021 - Eur Respiratory Soc
… Single-end reads of 50 bp length were produced with a minimum of 1 M reads per sample. …
k, m) Representative transmission electron microscopy of cultured control (k) versus CFTR …

Nanoblades allow high-level genome editing in organoids

V Tirolle, A Krug, E Bokobza, M Bulcaen, MM Ensinck… - bioRxiv, 2022 - biorxiv.org
Genome engineering has become more accessible thanks to the RNA programmable
endonucleases such as the CRISPR/Cas9 system. However, using this editing technology in …

Severity of the S1251N allele in cystic fibrosis is affected by the presence of the F508C variant in cis

…, M Ensinck, MS Carlon, M Boon, M Proesmans… - Journal of Cystic …, 2022 - Elsevier
Background In cystic fibrosis (CF), genotype-phenotype correlation is complicated by the large
number of CFTR variants, the influence of modifier genes, environmental effects, and the …