When 5-month-old Agustín Cáceres received his new gene on Dec. 17, 2010, correcting a severe immune deficiency, he was among the first patients to benefit from a rebirth of gene therapy, and the first patient to receive gene therapy at Boston Children’s Hospital. After steady improvements in technique, gene therapy represents a major shift in medicine, offering cures for conditions not previously treatable and for diseases often demanding heroic efforts. Since that historic day, a decade ago, we have treated more than 70 patients from 20 countries through 19 gene therapy clinical trials.
As the Gene Therapy Program enters its second decade, our team is optimistically embracing new gene-correction technologies such as gene editing and gene silencing. We feel fortunate to be at the forefront of this field with our patients and families providing motivation and purpose.
