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 ...

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 ...

Synthetic human chromosomes may transform gene therapy by carrying large therapeutic genes without altering natural DNA. Here ...

Researchers at Cincinnati Children's have demonstrated that AAV-mediated delivery of the human FMR1 gene can restore FMRP ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

The Ara Parseghian Medical Research Fund will provide financial backing for the multi-year research effort.

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

Children born profoundly deaf because of mutations in the OTOF gene are hearing speech, music, and everyday sounds after a single gene therapy infusion, and the gains are holding steady for up to 2.5 ...

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 ...

Some gene therapy treatments use modified adeno-associated viruses (AAVs) like the one shown here to deliver therapeutic genetic material into a patient’s cells. In a finding with implications for one ...