Publications by authors named "Zhichang Peter Zhou"
Article Synopsis
- Researchers are exploring gene replacement therapy using helper-dependent adenoviral vectors (HD-CFTR) to restore CFTR function in individuals with class I mutations where no functional protein is made.
- They found that HD-CFTR can significantly restore CFTR function in cultured nasal cells from CF patients, and the efficacy of this approach can help in testing other CFTR-modulating drugs.
Early efforts in cystic fibrosis (CF) gene therapy faced major challenges in delivery efficiency and sustained therapeutic gene expression. Recent advancements in engineered site-specific endonucleases such as clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 make permanent CF transmembrane conductance regulator () gene correction possible. However, because of safety concerns of the CRISPR/Cas9 system and challenges in delivery to inflamed CF airway, CRISPR-based gene correction strategies need to be tested in proper animal models.
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