The unprecedented study involves using the gene-editing technique CRISPR to edit a gene while it's still inside a patient's body. In exclusive interviews, NPR talks with two of the first participants.
Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, the treatment could change the lives of thousands of children with Duchenne muscular dystrophy.
The first gene therapy for hemophilia could be approved by the FDA within six months, according to the drugmaker, raising hopes among families. But the drug's price could be $3 million per patient.
Attempts to use the gene-editing tool CRISPR to develop a treatment for cancer seem safe and feasible in the earliest findings from the first three patients. "So far, so good," scientists say.
The latest advance is not only encouraging news for patients with severe combined immunodeficiency. It's a test case for all those scientists working to develop better gene therapy techniques.
Carl Zimmer wondered what secrets lurked in his genetic code — so he decided to have his genome sequenced. He writes about the implications of the study of genetics in She Has Her Mother's Laugh.
A small study finds promise for using gene therapy to treat patients with beta-thalassemia, a blood condition that can cause severe anemia. The experimental treatment is in early development.
The Food and Drug Administration approved Luxturna, a genetically modified virus that restores by ferrying a healthy gene into the eyes of patients born with a genetic disease that impairs sight.
After decades of hope and disappointment, doctors have now been able to treat several different types of genetic conditions by giving each patient a healthy version of their defective gene.
After many setbacks for genetic therapies, advisers to the Food and Drug Administration recommended approval of the first gene treatment for an inherited form of blindness.
The process modifies patients' immune cells to attack their own cancer cells. It was approved to treat acute lymphoblastic leukemia in children and young adults — the most common childhood cancer.
The latest accomplishment for gene therapy involves mice with inherited deafness. Meanwhile, the drugmaker Novartis is conducting the first trial of gene therapy for people with hearing loss.
By editing the genes in embryos in the lab, Chinese scientists showed that it's possible to change hereditary traits that cause a blood disorder. But the work also created unintended mutations.
