Articles related to GENE THERAPY

This phase 1–2 study of gene therapy in Crigler–Najjar syndrome patients offers promising insights into a novel treatment approach. Explore the detailed findings to understand how this therapy could revolutionize the management of this rare and severe condition.

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.

Discover how a groundbreaking trial using a new CRISPR gene-editing technique has successfully treated a patient with T-cell leukemia, a form of the disease resistant to conventional treatments. Learn how this innovative approach could revolutionize the treatment of various diseases affecting the blood, bone marrow, and immune systems.

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.