trial 2

In the early 2000s, a two-year-old girl was at risk of dying due to a genetic mutation causing heart failure. A man in his mid-thirties faced a similar fate with a genetic mistake destroying his brain. However, gene editing offers hope for treating and possibly curing genetic diseases. The breakthrough technology, CRISPR-Cas9, opened up new possibilities in genetic medicine. Gene editing is being explored for diseases like sickle cell anemia and inherited blindness, with some patients already experiencing improvement. In 2007, a breakthrough in gene therapy treated severe beta thalassemia by modifying the patient's bone marrow, resulting in healthy red blood cells. Gene editing is also being used in infectious disease research to develop new therapies and diagnostics. In the fight against Parkinson's disease, gene therapy is targeting the root cause by delivering special genes to the brain to produce dopamine and potentially slow disease progression. In early 2000s, the parents of a two-year-old girl tried that their daughter could die within the next year because a genetic mutation is causing her heart to fail. Time is quickly running out for me, writes a man in his mid-thirties, whose DNA harbors a genetic mistake certain to destroy his brain within a matter of years. That story about a man is a beacon of hope. It showcases the potential of gene editing not just for treating, but potentially curing genetic disease. Welcome to today's podcast, where we explore a thrilling question, can we cure genetic disease by rewriting DNA? This isn't just science fiction anymore, it's becoming a scientific reality with technologies like CRISPR, isn't it? Prepare to be amazed, Maha, because the story of gene editing goes way back to when science was just starting out. Back in the 1980s, scientists began tinkering with ways to edit genes, and since then, things have only gotten more exciting. The real game-changer came with CRISPR-Cas9, a breakthrough technology that opened up a whole new world of possibilities in genetic medicine. Imagine a future where genetic problems aren't so scary anymore, it's like fixing typos in a digital document. Nowadays, researchers are trying gene editing on all sorts of diseases, like sickle cell anemia and even some type of inherited blindness. It's still early days, but some patients are already feeling a lot better, thanks to these treatments. Hey, have you heard about the awesome gene therapy breakthrough back in 2007? They treated severe beta thalassemia by tinkering with the patient's bone marrow. Basically, they took out some blood stem cells, jazzed them up with a fancy gene called beta globin, using retroviral vectors, and voila! The patients started making healthy red blood cells again. What's even cooler is that 7 years later, they were still going strong, no more blood transfusions needed. How cool is that? In the world of infectious disease research, gene editing is taking center stage. Researchers are now using these techniques to craft new therapies, like tweaking immune cells so they can better spot and wipe out pathogens, like HIV and hepatitis B. Plus, CRISPR isn't just for editing genes, it's also revolutionizing how we detect pathogens with CRISPR-based diagnostics. We can now identify specific DNA or RNA sequences of virus and bacteria quickly and accurately detect them. This is the first step of CRISPR-based gene editing. You know, it's fascinating how gene therapy is stepping up in the fight against Parkinson's disease. Instead of just treating the symptoms, it's all about tackling the root cause head-on, which is the loss of those dopamine-producing brain cells. Picture this. They're using these clever retroviral vectors to smuggle in three special genes straight to the brain's hotspots. These genes are then fed to the patient's brain cells. The patient's brain cells are then fed to the patient's brain cells. They're using these retroviral vectors to smuggle in three special genes straight to the brain's hotspots. These genes basically give non-dopamine-producing cells a new superpower. They start churning out dopamine. It's like giving the brain a boost to regain muscle control and maybe even put brakes on disease progression. Pretty cool, huh?