Articles related to GENE THERAPY

Roctavian represents a turning point in hemophilia A management, offering patients like Maxwell a potential lifetime of relief from traditional therapies. This gene therapy directly targets the genetic cause, paving the way for innovative treatments in genetic disorders.

The therapy aims to restore progranulin protein levels in the brain, marking a significant step forward in the treatment of neurodegenerative disorders.

The novel finding that selective neuronal knock-out of STAT3 can potentially mitigate the progression of temporal lobe epilepsy underscores the necessity for further research into the JAK/STAT pathway’s involvement in epileptogenesis. This discovery may pave the way for more targeted, effective therapeutic strategies.

Treading the line between cost-effectiveness and equity in SCD management, gene therapy presents a paradigm shift. Curious about the economic equilibrium in this new era of treatment? Dive into the latest research insights.

Charu Aggarwal, MD, MPH, of the University of Pennsylvania’s Abramson Cancer Center in Philadelphia, H. Jack West, MD, of the City of Hope Comprehensive Cancer Center in the Los Angeles area, and Devika Das, MD, of the University of Alabama at Birmingham discuss the distinction between uncommon EGFR mutations and sensitizing mutations, as well as the lack of data on how to treat these patients with tyrosine kinase inhibitors.

The adenoviral vector–based gene therapy contains the gene for interferon alfa-2b. It is administered by catheter into the bladder once every three months. The virus penetrates the bladder endothelial cells and releases the gene, which is incorporated by the cells. As a result, the cells begin secreting high levels of the cancer-fighting interferon.