Archive for sickle cell disease
Could a pill treat sickle cell disease?
The new gene therapies for sickle cell disease — including the gene-editing treatment Casgevy, based on research at Boston Children’s Hospital — have been game-changing for the patients who have received them. But Stuart Orkin, MD, the Boston Children’s hematologist whose work led the way to Casgevy, wants to go even further. “The editing therapy ... Read More about Could a pill treat sickle cell disease?
A sickle cell first: Base editing, a new form of gene therapy, leaves Branden feeling ‘more than fine’
Though he doesn’t remember it, Branden Baptiste had his first sickle cell crisis at age 2. Through elementary school, he was in and out of the hospital with pain episodes, not knowing why. As he got older, he learned he had sickle cell disease. His red blood cells were forming sickle shapes and getting stuck ... Read More about A sickle cell first: Base editing, a new form of gene therapy, leaves Branden feeling ‘more than fine’
In a thriving gene therapy program, nursing leadership is the driving force
Gene therapy was made possible by decades of technological advances. But to execute gene therapy at scale? That would not be possible without the foresight, organization, and innovation of nursing leadership at Boston Children’s Hospital. The gene therapy implementation framework they have built has become a gold standard for the field. Founded in 2010, the ... Read More about In a thriving gene therapy program, nursing leadership is the driving force
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