“Big Pharmaceuticals” has been committed to producing a wide range of innovative drugs for many years. For them, gene therapy may be the place to give birth to the next “miracle.” Gene therapy, as its name implies, is a treatment that replaces defective genes with normal genes and has… Continue Reading Gene therapy is the leader, attracting countless giants to compete!

  In 1998, American scientists Andrew Fire and Craig Mello published a groundbreaking paper in Nature, confirming that double-stranded RNA (dsRNA) is the cause of post-transcriptional gene silencing (PTGS) in nematodes. They call this phenomenon RNA interference (RNAi).   The discovery of RNAi (Fig. 1) explains the confusing gene silencing… Continue Reading The world’s first RNAi drug “born” for 20 years and will “explode” in the next 10 years.

  Traditional gene therapy and emerging gene editing therapy for Duchenne Muscular Dystrophy (DMD), which one is better? Some recent research may give us some new thinking.   There are approximately 300,000 DMD patients worldwide. Due to the presence of mutations, patients are unable to produce dystrophin normally, causing atrophy… Continue Reading Traditional gene therapy vs. gene editing, who is the king of DMD treatment?

Recently, Dr. Scott Gottlieb, the US FDA’s director, issued a statement announcing that the FDA will continue to vigorously promote the development of gene therapy and released six new guidelines. Gene therapy was once a theory and has now become a therapeutic reality for patients. Gene therapy has the potential… Continue Reading Fully promote gene therapy, FDA released 6 new guidelines