Tag results:

cystic fibrosis

Evaluating CRISPR-Based Prime Editing for Cancer Modeling and CFTR Repair in Organoids

[Life Science Alliance] Investigators explored the use of prime editing in human organoids. Common TP53 mutations can be correctly modeled in human adult stem cell–derived colonic organoids with efficiencies up to 25% and up to 97% in hepatocyte organoids.

Response to Elexacaftor/Tezacaftor/Ivacaftor in Intestinal Organoids Derived from People with Cystic Fibrosis

[Journal of Cystic Fibrosis] The authors utilized an intestinal organoid approach to compare in vitro responses to two CFTR modulator drug combinations and to check potential inter-individual variability in therapeutic response to the triple combination.

Potential of Helper-Dependent Adenoviral Vectors in CRISPR-cas9-Mediated Lung Gene Therapy

[Cell & Bioscience] Scientists discuss the status of the application of CRISPR/Cas9 in cystic fibrosis gene therapy, the existing challenges in the field, as well as new hurdles introduced by the presence of CRISPR/Cas9 in the lungs.

Molecular Analysis of Pseudomonas aeruginosa Strains Isolated from Cystic Fibrosis Patients

[Scientific Reports] Scientists applied a multiple-locus variable-number tandem-repeat analysis for an efficient genotyping of clinical P. aeruginosa strains isolated from cystic fibrosis patients and compared results with a TRS-PCR typing.

Single-Cell-Resolved Differentiation of Human Induced Pluripotent Stem Cells into Pancreatic Duct-Like Organoids on a Microwell Chip

[Nature Biomedical Engineering] Researchers reported the single-cell characterization of the differentiation of pancreatic duct-like organoids from human induced pluripotent stem cells (hiPSCs) on a microwell chip that facilitated the uniform aggregation and chemical induction of hiPSC-derived pancreatic progenitors.

Vertex Announces National Reimbursement Agreement in France for KAFTRIO® and SYMKEVI® for Eligible Cystic Fibrosis Patients

[Vertex Pharmaceuticals, Inc.] Vertex Pharmaceuticals Inc. announced a national reimbursement agreement with the French Health Authorities for the cystic fibrosis medicines KAFTRIO® in a combination regimen with ivacaftor and SYMKEVIsup>®

Popular