People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid ...

"Moreover, the duration of follow-up is among the longest reported for a lentiviral vector-based gene therapy; 13 patients ...

If administered early, gene therapy has the potential to change the medical history of children born with metachromatic ...

Genome editing has advanced at a rapid pace with promising results for treating genetic conditions -- but there is always room for improvement. A new paper showcases the power of scalable protein ...

The two parties will leverage their respective strengths to accelerate the provision of this gene therapy to patients ... the commercialization of this research achievement overseas, with a ...

Personalized medicine, sometimes called precision medicine, is an emerging practice that uses an individual’s genetic profile to guide decisions about prevention, diagnosis and treatment of ...

Analysis from Precision Research estimates that advanced therapies including cell and gene therapy research will account for approximately 20% of the size of the total pharmaceuticals market. In 2023, ...

The therapy is designed to combat ocular problems including several pathologies of the cornea and glaucoma. This initiative is part of Genflow's continued efforts to optimize its gene therapy and ...

Even for those conducting the research, many uncertainties remain. “At present, not all hereditary rare diseases are potentially treatable with gene therapy,” explained Aiuti. “In choosing ...

In recent decades, the Triangle has developed into a hub for gene therapy treatments, through research at Duke University and UNC-Chapel Hill’s Gene Therapy Center as well as in the local ...

Ph.D., of the Center for Virology and Vaccine Research at BIDMC, member of the Gene and Cell Therapy Institute at Mass General Brigham, and member of the faculty at Harvard Medical School.

Gene therapy has emerged as a promising frontier in cystic fibrosis research, offering the potential to not just alleviate symptoms, but to address the genetic root of the disease itself. In this ...