Prenatal gene therapy has been used to prevent acute neuronopathic Gaucher’s disease, however this approach is using viruses to deliver normal copies of genes.
Scientists are taking advantage of the “self-homing” abilities of cancer cells and are creating armies of cancer-killing cells using CRISPR gene-editing.
A single administration of a therapeutic vector in mouse models cures type 2 diabetes and obesity in the absence of long-term side effects, researchers report.
New research links higher body fat with lower breast cancer risk before menopause.
Researchers at King’s College London have shown that rats with spinal cord injuries can re-learn skilled hand movements after being treated with a gene therapy.
Using genome-wide genetic approaches, researchers systematically screened for the loss of an additional gene that could rescue a genetic disease, and found it.
FDA commissioner, Scott Gottlieb said that he expects the FDA to approve 40 gene therapies by 2022 in a fireside chat at the 2018 BIO International Convention.
Singapore could become the second country to legalise MRT but they are considering a new method that differs from the two currently legal in the UK.
In a new multicenter clinical trial, a personalised vaccine improved the survival rates of some people with the deadly brain cancer glioblastoma.