Archive for gene therapy
Restoring muscle function in a rare, devastating disease: Part 1
Part 1 of a two-part series. (Read part 2.) Sixth-grader William Ward cruises the hallways at school with a thumb-driven power chair and participates in class with the help of a DynaVox speech device. Although born with a rare, muscle-weakening disease called X-linked myotubular myopathy, or MTM, leaving him virtually immobile, he hasn’t given up. Neither has Alan Beggs, ... Read More about Restoring muscle function in a rare, devastating disease: Part 1
Gene therapy: Two years and holding
For the Cáceres family of Argentina, it’s a joyous holiday homecoming. Agustín, who received gene therapy at 5½ months of age, journeyed with his family to Boston for a check-up and got a clean bill of health. Agustín was born with the rare immune-deficiency disorder SCID-X1. More popularly known as “bubble boy” disease, it left him defenseless against ... Read More about Gene therapy: Two years and holding
Gene-therapy trial will attempt to restore hearing in deaf mice
Sound waves produce the sensation of hearing by vibrating hair-like structures on the inner ear’s sensory hair cells. But how this mechanical motion gets converted into electrical signals that go to our brains has long been a mystery. Scientists have believed some undiscovered protein is involved. Such proteins have been identified for taste, smell and ... Read More about Gene-therapy trial will attempt to restore hearing in deaf mice
Avoiding the needle: Engineering blood vessels to secrete drugs
People who rely on protein-based drugs often have to endure IV hookups or frequent injections, sometimes several times a week. And protein drugs – like Factor VIII and Factor IX for patients with hemophilia, alpha interferon for hepatitis C, interferon beta for multiple sclerosis — are very expensive. What if they could be made by people’s own ... Read More about Avoiding the needle: Engineering blood vessels to secrete drugs
A new start for gene therapy for ‘bubble boy’ disease: First U.S.-treated patient doing well
Until this month, Agustín Cáceres’s baptism was the only time his family could come close to him. Everyone had to wear masks, gloves and gowns. After that, he went into isolation, along with his mother Marcela, who came out only for meals. His father Alberto, and his four-year-old brother Jeremías, kept to a separate bedroom. ... Read More about A new start for gene therapy for ‘bubble boy’ disease: First U.S.-treated patient doing well
Moving gene therapy into high gear
Gene therapy, still experimental but beginning to enter the clinic, attempts to utilize advanced molecular methods to treat and even reverse genetic diseases. The field started in earnest about 25 years ago and has had many setbacks along the way to its recent earliest successes. International collaboration has been critical. Children’s Hospital Boston is one ... Read More about Moving gene therapy into high gear