Gene therapy is an experimental technique that uses the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease. A variety of non-viral and viral vectors have been developed for gene therapy. How do they work? Is gene therapy safe?
Precision medicine is an evolving therapeutic approach in health care industry, particularly for cancer care. Recently, this approach is also being considered for neurodegenerative diseases and other rare diseases. Though, focus of this article is Precision medicine – ...Learn More
The emerging CRISPR technology has expanded the scope and applications of gene therapy. Translation of CRISPR-based gene editing to in vivo application however, is still not easy. A vital challenge is to develop safe, efficient, and clinically suitable in vivo delivery approaches ...Learn More
Nature Reviews Drug Discovery published an article "Gene-editing pipeline takes off" on May 15, 2020, summarized the clinical trials of genome-editing agents, including CRISPR-Cas9 editors, zinc finger nucleases and TALENs, for ex vivo, immuno-oncology and in vivo treatment. It's ...Learn More
In March 2020, the European Medicines Agency recommended granting a conditional marketing authorisation in the European Union for a virus vector-based gene therapeutic drug Zolgensma (ema, 2020). The drug have been used before in United States and Japan for the treatment of ...Learn More
Gene therapy is the treatment of disease by rebuilding defective genetic material (Ermak G, 2015). The whole process involves identifying abnormal gene, synthesizing and delivering identical healthy gene into a cell, tissue, or whole organ, with the goal of curing a disease or at ...Learn More