The CRISPR tool is capable of repairing the genetic defect responsible for the immune disease chronic granulomatous disease. However, researchers have now shown that there is a risk of inadvertently ...
This is because they can be used to correct specific defective sections of the genome. Unfortunately, however, there is a catch: under certain conditions, the repair can lead to new genetic defects – ...
Ensoma, a genomic medicines company advancing the future of medicine through one-time, in vivo treatments capable of precisely and durably engineering the hematopoietic system, today unveiled its ...
More information: Vera Bzhilyanskaya et al, High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease, Science Translational Medicine (2024).
The funds will enable Somagenetix to advance its leading gene therapy-candidate, SGX-001, into a clinical trial for the treatment of p47 Chronic Granulomatous Disease (CGD). CGDs are a group of ...
You may also notice that you are more sensitive to light, feel like there is something in your eye, or have vision changes. To improve symptoms, your eye care provider may recommend: Granulomatous ...