Search Results for: gene therapy
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 …Read More
Making ionocytes: A step toward cell or gene therapy for cystic fibrosis
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators were a breakthrough for cystic fibrosis, improving the movement of chloride and water …Read More
Making history: Gene therapy for CCALD gives Conner a second chance
Like a lot of 6-year-olds, Conner Hess finds joy in simple acts: drawing pictures, cuddling his two cats, and playing …Read More
Could gene therapy relieve post-hemorrhagic hydrocephalus?
Premature infants, especially very low birthweight babies, are at risk for intraventricular hemorrhage. A frequent complication of these brain bleeds …Read More
Matthew, the ‘wee marvel’: One of the first ALD gene therapy recipients
When the Elliott brothers are asked how many siblings they have, they always say, “four.” It’s a way of honoring …Read More
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 …Read More
Teaming up to fast track a novel gene therapy for Diamond-Blackfan anemia
Looking at Lindsay Krieg, it’s impossible to tell she has spent her entire 29 years in an out of hospitals …Read More
After decades of evolution, gene therapy arrives
As early as the 1960s, scientists speculated that DNA sequences could be introduced into patients’ cells to cure genetic disorders. …Read More
A rebirth in Boston: Gene therapy turns 10
Lea la versión en español. Dec. 17 marks a decade since Agustín Cáceres was “renacido” — reborn. That’s how his …Read More
Hemophilia A gene therapy paves the way for a life with less pain
Matthew Harrington, a 35-year-old oil-field worker from Douglas, Wyoming, knows pain. Born with hemophilia A, a rare condition in which …Read More