gene therapy

PRINT, a new gene therapy technique, employs bird-derived retrotransposons to insert whole genes into a safe zone of the human genome, offering a complementary approach to CRISPR-Cas9 by potentially enabling the treatment of diseases without the risk of gene disruption or cancer. Credit: SciTechDaily.comRetrotransposons can insert new genes into a “safe harbor” in the genome, complementing CRISPR gene editing.The recent greenlighting of a CRISPR-Cas9 treatment for sickle cell disease underscores the efficacy of gene…

An illustration of chimeric antigen receptor (CAR)-T cells attacking a cancer cell.Illustration: ART-ur (Shutterstock)A relatively new cancer treatment known as CAR T cell therapy is perhaps more dangerous than we knew. The Food and Drug Administration found evidence that the treatment can potentially raise the risk of secondary cancers and is telling makers of these products to add a boxed warning detailing the risk to their labeling. However, the FDA still says that the positives of CAR T therapy outweigh the potential…

To get the new genetic material into cells, they engineered harmless viruses to carry it. Doctors carefully injected a tiny amount of liquid containing the viruses into a part of the children’s inner ears called the cochlea, a spiral-shaped chamber that contains hair cells. The first patient in the trial received the gene therapy in December 2022. Researchers followed the participants, who ranged in age from 1 to 6 years old, for 23 weeks after treatment.While the gene therapy did not give the children a “normal” level of…

December 28, 20233min readHere’s the best, most inspiring and coolest science we encountered this yearBy Sarah Lewin Frasier Scientific American’s reporting has played a crucial role in helping readers understand the wild ups and downs of 2023—simply look at our health roundup and climate roundup as proof of that. But if you’re looking for good vibes only heading into 2024, don’t worry: Scientific American has you covered. We’ve gathered 10 of the most amazing breakthroughs, examples of awe-inspiring research and just…

An example of what a deep brain stimulation device looks like under X-ray imaging.Photo: Hellerhoff/Wikimedia CommonsThe idea of using electricity to treat mental illness has understandably come with some stigma, given the grim and sometimes abusive history of “shock therapy” in the earliest days of psychiatry. But in the modern day, various methods of brain stimulation have shown real promise in improving depression and other illnesses that otherwise looked untreatable. These treatments, it’s theorized, can somewhat…

December 19, 20235min readFrom new uses for weight-loss drugs to the first CRISPR gene editing therapy, these were some of the most impactful health stories of the yearBy Tanya LewisIn an operating room at the University of Maryland Medical Center on September 20, 2023, surgeons transfer a genetically modified pig heart to the operative field for transplant. In world with so much urgent and depressing news, it can be hard to take a moment and reflect on all of the encouraging scientific and medical progress that has…

Our B cells help prevent us from getting sick. Their job is to make antibodies, immune system proteins that fight off viruses and other foreign invaders. And they make a lot of antibodies—thousands of them every second. What if these antibody factories could be harnessed to make other things the body needs?That’s the idea behind a trial launched by Seattle-based biotech company Immusoft. The company announced today that its scientists have genetically programmed a patient’s B cells and put them back in his body in an…

CRISPR, the gene-editing technology that has revolutionized biological research, is finally available as a medical treatment with regulatory approval. On December 8 the U.S. Food and Drug Administration approved the first CRISPR treatment for sickle cell disease.The treatment, called exa-cel and made by the companies Vertex and CRISPR Therapeutics, edits a gene involved in red blood cell shape and function. It appears to functionally cure the disease for at least one year. The FDA’s decision makes the U.S. the second…

Recent research suggests that stem cell gene therapy, utilizing CRISPR-Cas9 technology, offers a promising curative treatment for sickle cell disease, marking a significant advancement in the field of gene therapy. New findings from a clinical trial, recently published in the New England Journal of Medicine, suggest that stem cell gene therapy could be a potential curative treatment for sickle cell disease (SCD), a painful, inherited blood disorder. This research adds to the growing evidence that supports gene therapy as…

Scientists use harmless viruses to ferry and insert the new genetic material because of their natural ability to get inside cells. But the potential for these viruses to accidentally trigger another cancer has long been considered a theoretical risk. In its notice, the FDA said the use of these viruses may have played a role in patients developing secondary cancers.The downside of using viruses is that they tend to drop off their genetic cargo at a random place in a person’s genome. Depending on where this new genetic…