Archive for sickle cell disease
Sickle cell gene therapy and boosting fetal hemoglobin: A 75-year history
Ed. Note: This post updates an earlier post from 2018. In a landmark decision today, the Food and Drug Administration (FDA) approved two gene therapies for sickle cell disease. One of them, Casgevy, has deep scientific roots at Boston Children’s Hospital — and is also the first therapy using CRISPR gene editing to gain FDA ... Read More about Sickle cell gene therapy and boosting fetal hemoglobin: A 75-year history
Sickle cell disease, gene therapy, and blood cancers: Mysteries remain
Gene therapy trials for sickle cell disease have been showing great promise, even offering hope of a cure. But in early 2021, the trials ground to a halt after reports of blood malignancies in two people in a trial sponsored by bluebird bio. Investigations later concluded that the gene therapy delivery vectors were likely not ... Read More about Sickle cell disease, gene therapy, and blood cancers: Mysteries remain
How growing up with sickle cell disease is shaping Nancy’s future
Imagine appearing completely healthy while managing a life-threatening condition. Most people who meet Nancy Blankson would never know she has sickle cell disease (SCD). Her symptoms are not easily visible. It’s a challenge she lives with every day along with periodic pain crises, which at times, she says, can be virtually unbearable. Nevertheless, the 20-year-old ... Read More about How growing up with sickle cell disease is shaping Nancy’s future
Transitioning to adult care for sickle cell disease: Ariyanna’s journey
Ariyanna Agnew sits in a waiting room at Beth Israel Deaconess Medical Center (BIDMC). The 22-year-old, who has been a patient of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Sickle Cell Disease Program for more than a decade, is seeing an adult hematologist for the first time. It’s a milestone event that would ordinarily ... Read More about Transitioning to adult care for sickle cell disease: Ariyanna’s journey
Getting COVID-19 vaccines to medically fragile children
As COVID-19 vaccines slowly roll out, should children who need complex care or have serious medical conditions be vaccinated? We asked experts around Boston Children’s Hospital, and their resounding answer is: Yes, if they are age 16 or older. Boston Children’s is actively planning to make vaccination happen for as many patients as possible, as ... Read More about Getting COVID-19 vaccines to medically fragile children
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 about Gene therapy’s future may be all about the bases
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 about Leaning into Sydney: A team approach to renovascular hypertension
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 about Decoding sickle cell disease offers new outlook for Lamarcus
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 about Gene therapy to boost fetal hemoglobin continues to do well in sickle cell trial
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 about Manny: Hoping new research helps others with sickle cell disease