Search Results for: gene therapy
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
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 …Read More
Gene therapy with a new base editing technique restores hearing in mice
Using a new genetic engineering technique, known as base editing, researchers from Boston Children’s Hospital and the Broad Institute of …Read More
Gene therapy reverses heart failure in mouse model of Barth syndrome
Barth syndrome is a rare metabolic disease caused by mutation of a gene called tafazzin or TAZ. It can cause …Read More
Good early results with gene therapy for rare immune deficiency
Brenden Whittaker, a college student in Ohio, has been caught off guard by his good health. Since he was young, …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 …Read More
Sofie’s story: A new gene therapy treatment for SMA
Sofie Petrovická sits up in the exam room, smiling and laughing with her physical therapist, Elizabeth Maczek. Though she’s just …Read More
Tissue models and a gene therapy for a deadly heart arrhythmia
Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a leading cause of sudden death from cardiac arrest in children and young adults. …Read More
Our SCID gene therapy journey
In just 18 months, our son Paul Gallagher has been through more than what most people go through in a …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 …Read More