News

Boston Children’s Hospital to receive $1.5M to initiate basic work to expand gene therapy treatment of sickle cell disease into developing countries

The research will focus on adapting gene therapy for use in low-resource areas with high rates of the disease.

New gene therapy strategy for sickle cell disease shows early promise in humans

A patient with a severe form of sickle cell disease is symptom-free after gene therapy knocks down BCL11A and restores fetal hemoglobin production.

A new gene therapy transplantation technique could improve treatment of neurodegenerative disease

Alessandra Biffi, MD, led a study that shows the efficacy of a new gene therapy transplantation technique that could have future applications for treating neurodegenerative diseases.

New gene therapy clinical trial offers hope for sickle cell disease

David Williams, MD, discusses Dana-Farber/Boston Children's new gene therapy clinical trial for sickle cell disease in a live webchat.

Gene therapy halts progression of cerebral adrenoleukodystrophy in clinical trial

The devastating neurodegenerative disease typically affects young boys and causes death within 10 years

The space between heartache and happiness: Two sons with adrenoleukodystrophy and a gene therapy clinical trial

For the Rojas family, a clinical trial of gene therapy for adrenoleukodystrophy has meant both happiness and heartache.

Vietnamese boy thrives after gene therapy for Wiskott-Aldrich syndrome

After being diagnosed with Wiskott-Aldrich syndrome, Duy Anh "Harry" Le traveled with his family to Boston for a gene therapy clinical trial, and is now a happy and healthy two-year-old.

Gene Therapy 2.0

Levi Looks, diagnosed through newborn screening with “bubble boy” disease, is a healthy toddler thanks to gene therapy, resurgent now after early setbacks, MIT Technology Review reports.

One boy’s cure raises hopes and questions about gene therapy for sickle cell disease

Dana-Farber/Boston Children’s researchers, who are developing a gene therapy trial for sickle cell disease, commented in Stat and other outlets on a case report from a different trial.

Gene therapy trial gives Coopersville toddler second chance at life

A West Michigan toddler born with “bubble boy” disease is active and healthy, thanks to newborn screening and gene therapy, Fox 17 TV in Grand Rapids reports.