Scientists looked at multiple techniques used to measure the modified viruses deployed in some gene therapy research and treatments. One technique, known as SEC-MALS, was the most precise and accurate ...

Some of the most expensive drugs currently in use are gene therapies to treat specific diseases, and their high cost limits their availability for those who need them. Part of the reason for the cost ...

In a finding with implications for one of the most promising cutting-edge medical treatments, researchers evaluated several measurement techniques commonly used in gene therapy. The study determined ...

TREATING diseases by delivering genes to tissues in the body remains a huge challenge. But a non-invasive technique for monitoring the activity of added genes should help improve gene therapy methods.

Analyzing the purity of adeno-associated virus (AAV)-based gene therapy products requires effective methods for detecting host proteins, DNA, and incomplete viral particles in samples. It is a ...

Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person's genome, often with enough variation that even two individuals who share the same ...

Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying gene cargoes because of their superior gene segmentation flexibility and ...

What if we could repair fundamental parts of our bodies? Gene therapy promises just that. It's like having a molecular mechanic that can fix or replace faulty genes, potentially curing diseases ...

A recent Harvard study introduces a new method for treating spinal muscular atrophy. The study used "base editing" to target the disease's root cause by modifying the survival motor neuron gene. For ...

Gene therapy is still in development to test its safety and effectiveness, but it may be a promising new treatment for rheumatoid arthritis (RA). Gene therapy alters certain genes to help treat ...