Gene therapy for CF.

Gene therapy for CF.

Background: Transcription factors regulate gene expression by interacting with their specific DNA binding sites. Some transcription factors, particularly those involved in transcription initiation, always bind close to transcription start sites (TSS). Others have no such preference and are functional on sites even tens of thousands of base pairs (bp) away from the TSS. The Cyclic-AMP response e...

CF-GeNe: Fuzzy Framework for Robust Gene Regulatory Network Inference

Most Gene Regulatory Network (GRN) studies ignore the impact of the noisy nature of gene expression data despite its significant influence upon inferred results. This paper presents an innovative Collateral-Fuzzy Gene Regulatory Network Reconstruction (CF-GeNe) framework for Gene Regulatory Network (GRN) inference. The approach uses the Collateral Missing Value Estimation (CMVE) algorithm as it...

Temporal regulation of CFTR expression during ovine lung development: implications for CF gene therapy.

The cystic fibrosis transmembrane conductance regulator (CFTR) protein is a small conductance chloride ion channel that may interact directly with other channels including the epithelial sodium channel (ENaC). CFTR is known to be more abundant in the airway epithelium during the second trimester of human development than after birth. This could be a consequence of the change in function of the ...

ژن درمانی Gene Therapy

Ivacaftor Therapy in CF Patients: Single Center Experience

Ivacaftor is the first novel cystic fibrosis pharmaceutical that acts at the molecular level to potentiate cystic fibrosis transmembrane conductance regulator (CFTR) function and was first approved for clinical use in 2012. We are sharing our single center experience of five patients: four from pediatric age group and one adult patient. All patients had both subjective and objective improvement...