In Vivo Gene Articles & Analysis
7 articles found
Gene therapy is a process where a new, correct version of the CFTR gene is placed into a person's cells. ...
In many cases, gene therapy requires a vector to deliver the gene therapy drug to the target cell. ...
The Clinical CRISPR Successes Are Stacking Up. But Where’s the Emphasis on Quality Control? In mid-September, Intellia released some promising data on two of their CRISPR-based therapeutics. These results are a significant milestone for genome editing and validation of its effectiveness in the clinic. However, the safety of CRISPR-based therapeutics has been a major concern and part of a ...
This was the first clinical trial result of an in vivo CRISPR gene editing therapy that has been published. The results of the clinical trial were published in the New England Journal of Medicine, a leading international medical journal, under the title: CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. The study ...
and what makes them good candidates for gene therapy? Introduction In the first part of our three part series introducing AAV as a gene therapy vector, we talked about basic AAV vector biology. ...
We investigated the endocrine‐disrupting effects of PFOS using a combination of in vitro and in vivo assays. Reporter gene assays were used to detect receptor‐mediated (anti‐)estrogenic, (anti‐)androgenic, and (anti‐)thyroid hormone activities. The effect of PFOS on steroidogenesis was assessed both at hormone levels in the supernatant and at expression levels of ...
Here we report the improvement of the recombinase-based in vivo expression technology (R-IVET) by the implementation of two additional reporter genes. The first one is an -galactosidase gene (melA), which facilitates the rapid identification of in vivo-induced genes. Second, the bacterial luciferase ...