Boston Children’s Hospital is an international leader in gene therapy, with one of the largest and most experienced gene therapy programs in the world. Founded in 2010, our Gene Therapy Program offers patients clinical expertise, pioneering research, and a commitment to caring for rare and difficult-to-treat conditions.
Our dedicated infrastructure sets us apart: Our core team works in close collaboration with disease experts at Dana-Farber Cancer Institute. Our efficient enrollment process allows children to move smoothly and quickly from enrollment to treatment. As gene therapy continues to evolve, our team adopts new gene-correction technologies and conducts ongoing research to make these techniques more precise and safer than ever.