Archive for sickle cell disease
Gene therapy’s future may be all about the bases
Gene therapy offers the possibility of a cure for many genetic disorders, especially those involving a single gene. The first kind of gene therapy used a virus to carry a corrected copy of the gene into people’s cells. When the early viral vectors used in the 1990s were found to have off-target effects, sometimes even ... Read More
Leaning into Sydney: A team approach to renovascular hypertension
In most ways, Sydney Murphy is a typical 3-year-old girl: She watches the movie The Princess and the Frog on repeat, names most of her dolls after the main character, Tiana, and loves the color pink. But she’s also wise beyond her years. “She knows how to use a blood pressure cuff and is really ... Read More
Decoding sickle cell disease offers new outlook for Lamarcus
When Lamarcus Jean visits the Hematology Clinic at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, he makes himself right at home. The 6-year-old, whom his mom, Stephanie, describes as “wise beyond his years,” has been a patient here since he was born. Lamarcus has sickle cell disease, an inherited blood disorder caused by a mutation ... Read More
Gene therapy to boost fetal hemoglobin continues to do well in sickle cell trial
A pilot gene therapy treatment for sickle cell disease, restoring patients’ ability to make fetal hemoglobin, has produced good results in the first three patients to receive it. Investigators at Boston Children’s Hospital reported the findings of their ongoing clinical trial this week at the American Society of Hematology (ASH) annual meeting. The three adult ... Read More
Manny: Hoping new research helps others with 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 7-year-old Aiden, himself and others living with the inherited blood disorder. ... Read More
Reviving fetal hemoglobin in sickle cell disease: First patient is symptom-free
Manny Johnson of Boston, 21, previously required monthly blood transfusions to keep his severe sickle cell disease under control. After receiving a new gene therapy treatment, he’s been symptom-free for six months. Researchers at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reported Manny’s case Saturday at the American Society of Hematology meeting in San Diego. Manny ... Read More
Sickle cell gene therapy to boost fetal hemoglobin: A 70-year timeline of discovery
Boston Children’s Hospital is now enrolling patients age 3 to 35 in a clinical trial of gene therapy for sickle cell disease. Based on technology developed in its own labs, it differs from other gene therapy approaches by having a two-pronged action. It represses production of the mutated beta hemoglobin that causes red blood cells to ... Read More
BCL11A-based gene therapy for sickle cell disease passes key preclinical test
Research going back to the 1980s has shown that sickle cell disease is milder in people whose red blood cells carry a fetal form of hemoglobin. The healthy fetal hemoglobin compensates for the mutated “adult” hemoglobin that makes red blood cells stiffen and assume the classic “sickle” shape. Normally, fetal hemoglobin production tails off after birth, shut ... Read More