New Research Effort Aims to Improve Treatment for Sickle Cell Disease

Emmanuel “Manny” Johnson, Jr., shares many loves with his little brother, Aiden—from basketball to video games. One thing he wishes they did not share is sickle cell disease (SCD), so Manny is playing a role in a new effort to improve treatment for patients like seven-year-old Aiden, himself, and others living with the inherited blood … Continued

How Gene Therapy Works in Treating Cancer and Other Conditions

There are many types of gene therapy, but all of them involve adding to or modifying the genetic program in a person – his or her DNA blueprint that provides instructions for building and operating the body. Gene therapy technology has been developed over several decades and has been successfully used to correct rare blood … Continued

CRISPR Enables Cancer Immunotherapy Drug Discovery

This originally appeared on Vector, Boston Children’s Hospital’s blog. A novel screening method using CRISPR-Cas9 genome editing technology has revealed new drug targets that could potentially enhance the effectiveness of PD-1 checkpoint inhibitors, a promising new class of cancer immunotherapy. The method, developed by a team at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, uses CRISPR-Cas9 … Continued

Gene Therapy Helps Treat Boy’s Rare Immune Disorder

In their Brookline home-far-away-from-home, 2-year-old Duy Anh “Harry” Le plays with blocks and pop-up toys on the floor with his mother, Thao Nguyen. He is lively and happy, and his skin is clear. He looks almost nothing like the sickly baby covered in eczema who arrived in Boston from his native Vietnam in November 2016 … Continued

Blood Relatives: Family Bound by Love and Rare Blood Disorder

Tracy Antonelli was four when she was diagnosed with thalassemia, a rare blood disorder that occurs often enough in Mediterranean countries like Italy that an old adage, uttered only partially in jest, warns Italian-Americans against marrying other Italian-Americans. In 2002, a grown-up Antonelli wed Patrick Mooty, whose background is mostly Irish. Their three daughters – … Continued

How Pediatric and Canine Cancer Similarities Can Help Both Children and Pets

In March 2016, Ollie the pug, a therapy dog at Boston Children’s Hospital, paid a bedside visit to 7-year-old Carter Mock. Both dog and boy lost limbs to osteosarcoma, a cancer of the bone. Ollie’s left front leg was amputated at the shoulder. After removing the tumor in Carter’s left leg bone, surgeons fashioned  a … Continued

Months After Transplant, Teen Hits the Soccer Field

Some 100 days after receiving a stem cell transplant to cure his severe aplastic anemia, 13-year-old Behaylu Barry still couldn’t invite friends into his home. He can’t return to school until January, when his immune system will finally be strong enough to fight the pathogens present in indoor spaces. Yet  Behaylu was doing so well … Continued

Faces of Childhood Cancer: Steven Clifford

Steven Clifford is an 18-year-old osteosarcoma survivor. A Boston native, he starts college at the University of California, San Diego this month. This is his story. Life is made up of many difficult decisions. However, imagine my surprise when I had to make a tough and potentially life changing decision at the young age of … Continued