In a recent article published in Genetic Engineering News, the CRISPR technology is highlighted as a tool for clinical gene therapy in humans. In February of this year, the first patient was dosed with a CRISPR-Cas9 therapeutic in a Phase I/II trial for treatment of β-thalassemia (a type of inherited blood disorder). However, before CRISPR can become a broad-spectrum treatment for all genetic disorders, tests in animal models must first be conducted to ensure efficacy and safety. Taconic Biosciences' Dr. Caroline Horizny is cited as stating that "Studies published in Science and Nature have demonstrated the application of genetically humanized mouse models to testing both the efficacy and delivery method of gene therapy in vivo. These models will help to enhance understanding of CRISPR-based gene therapy and advance its use in disease treatment:
"Gene therapy, like a car with mechanical problems, has a history of jerking to life and then quickly stalling. Fortunately, gene therapy has the benefit of a kind of roadside assistance, one that comes in the form of gene editing technology, which is becoming more precise. It can help gene therapy run more smoothly. For example, gene editing can now be used to silence or repair a faulty gene, rather than insert an entire gene into the genome. (If a replacement gene is poorly placed, it can cause insertional mutagenesis.) Several forms of high-precision gene editing can give gene therapy a jump, but the most electrifying form is probably CRISPR."
Read the complete article at: genengnews.com