Germline gene therapy and somatic gene therapy are two ... is often at the center of objections to the procedure. So far, only a few studies of this potential have been published, and none have ...
Since two gene therapies were approved for sickle cell disease in late 2023, only a handful of patients have started the ...
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 ...
National Institute of Standards and Technology scientists looked at multiple techniques used to measure the modified viruses ...
The website you are visiting is protected and accelerated by Incapsula. Your computer may have been infected by malware and ...
Common measurements for modified adeno-associated viruses (AAVs), a gene therapy delivery method, showed substantial ...
Swiss drugmaker Novartis said on Monday its gene therapy helped improve motor function in children with a rare muscle ...
Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene ...
Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?
On 30 December 2024, Novartis reported that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) reached ...
And as one would expect or hope to see in a gene therapy trial, there was a significant and marked reduction in the use of factor.” Moving forward, he said, the message to clinicians is that ...