CRISPR Gene Therapy Cures Childhood Genetic Diseases, by Doc Smo and Sonya Corina Williams (Pedcast)

  If you know a child who has an incurable genetic disease like sickle cell anemia, cystic fibrosis, or muscular dystrophy…there is good news and it’s called CRISPR technology. Stay tuned to learn more about this incredible technology.   Musical Intro   Medical Science Takes a Leap Forward When we look back at the history of medical science, there are notable moments when technology or treatments jump forward in leaps and bounds, usually as a result of a new technology. Good examples are the development of the smallpox vaccine in 1796 and the discovery of the antibiotic penicillin, in 1928. In the last few years, there has been another one of these breakthroughs - it’s called CRISPR. In fact, Jennifer Doudna and Emmanuelle Charpentier shared the Nobel prize in Chemistry in 2020 for their discovery and application of CRISPR  gene editing. In a moment, you will see why.   What Exactly is CRISPR? So, what is CRISPR? The catchy acronym is short for a not-so-catchy mouthful of a name: “clustered regularly interspaced short palindromic repeats”. Think of CRISPR as a genetic “cut” and “paste” tool, cutting out disease causing genes and replacing them with healthy normal genes. DNA scissors of a sort. With CRISPR, you can substitute a specific sequence of a child’s disease producing DNA and replace it with a different, properly functioning, sequence. Move over Dr. McCoy of Star Trek, humanity is about to go where no man has ever gone.   Why is this so revolutionary? Because it offers the potential to cure congenital genetic disorders that plague children. With CRISPR we are not treating disease we are eliminating it! When it works, it converts dysfunctional cells into their normal healthy functioning cousins.   Target #1- CRISPR Aims to Cure Sickle Cell Disease   Not only is this technology revolutionary but it already been successful. CRISPR was used for the first time in a human sickle cell anemia patient in 2019. For those not familiar with sickle cell anemia, it is