Cystic Fibrosis (CF) is an autosomal recessive disorder due to mutations in the CF
transmembrane conductance regulator (CFTR) gene that lead to defective ion transport in …

Shortly after the cystic fibrosis (CF) gene was identified in 1989, the race began to develop a
gene therapy for this condition. Major efforts utilized full-length cystic fibrosis transmembrane …

The present invention provides new compositions and meth ods for preventing and treating
pathogen infection. In particu lar, the present invention provides compounds having an …

The CRISPR-Cas9 system is attractive for gene therapy, as it allows for permanent genetic
correction. However, as a new technology, Cas9 gene editing in clinical applications faces …

Recombinant adeno-associated viral (rAAV) vectors are being evaluated in animal models
and humans. Pre-clinical data demonstrating vector safety, efficiency and efficacy have been …

The application of adeno-associated virus (AAV) vectors to cancers is limited by their low
transduction efficiency. Previously, we reported that γ-ray enhanced the second-strand …

Adeno-associated virus (AAV) is currently being used in several human gene therapy trials,
including one targeting hemophilia B and another targeting cystic fibrosis, and to date has …

Background Cystic fibrosis is caused by a defective gene encoding a protein called the
cystic fibrosis transmembrane conductance regulator (CFTR), and is characterised by …

An increasing number of strategies for molecular treatment of disease rely on the adeno-
associated virus (AAV) as a therapeutic gene delivery vector. One of the most attractive …

Polyethylenimine (PEI) is a well‐known cationic polymer which has high transfection
efficiency due to its buffering effect. However, nondegradability, cytotoxicity, aggregation …