Researchers have reported promising results from two gene therapy trials for the treatment of two different rare diseases. The team used lentiviral vectors to swap out faulty genes for working copies in cultures of the patients’ own hematopoietic stem cells (HSCs)—cells that mature into all different types of blood cells—before transfusing the corrected cells back into the patients.
In the both trials, 18–32 months after treatment a high percentage of the patients’ blood cells were genetically corrected, and disease progression was arrested.
Until recently clinical application of the approach has been held back by problems with gene-transfer efficiency and safety.
Read the full article here: Gene Therapy Coming of Age?
- “Defanged HIV Shows Promise in Gene Therapy,” Wall Street Journal
Read about a recent study in which HIV was used as a vector for gene therapy to guard against a genetic neurological disorder.
- “Gene therapy’s time seems to have come,” Boston Globe
Gene therapy isn’t just providing positive results in the literature. It’s also becoming increasingly available.