Gene therapy is a revolutionary approach for treating severe genetic diseases, typically involving the replacement or repair of faulty genes. Currently, the most effective delivery system is based on adeno-associated virus vector,Read More…
Recently, a CRISPR-Cas9 therapy for sickle cell disease has been approved, showcasing the potential of gene editing tools to effectively knock out genes for treating genetic disorders. However, the replacement of entireRead More…
In a new study, researchers from University College London have developed a novel gene therapy to treat a devastating form of childhood epilepsy, showing significant reduction in seizure episodes in mouse models.Read More…
Recently, researchers from the Utrecht University Medical Center published a research paper in the Nature subsidiary journal, Nature Cardiovascular Research, titled: “Therapeutic efficacy of AAV-mediated restoration of PKP2 in arrhythmogenic cardiomyopathy.” TheRead More…
In 50 families from the Netherlands, the United Kingdom, the United States, and China, each family has a child paralyzed due to a mutation in the Cntnap1 (Contactin-Associated Protein 1) gene. TheseRead More…
Researchers from Ohio State University, Oregon Health and Science University, Wake Forest School of Medicine, and the University of California, San Francisco, have published a research paper in the prestigious medical journalRead More…
Innovative gene therapy may hold the key to treating chronic pain in humans by reducing sodium ion levels, a recent study published in the international journal “Proceedings of the National Academy ofRead More…
Hepatocellular carcinoma (HCC), caused by increased obesity levels, poses a new health burden. However, there is currently a lack of optimal therapies for treating HCC. Tyrosine kinase inhibitors, including sorafenib, lenvatinib, regorafenib,Read More…
The CRISPR-Cas system, a DNA recognition tool, has rapidly advanced as a means of editing cellular DNA. In the engineered version, a single guide RNA (gRNA) guides Cas9 to the target sequenceRead More…
Hemophilia is a genetic disorder characterized by the deficiency of clotting factors, resulting in spontaneous or excessive bleeding during surgery or trauma. Hemophilia B is caused by the deficiency of clotting factorRead More…