Archive for gene therapy
BCL11A-based gene therapy for sickle cell disease passes key preclinical test
Research going back to the 1980s has shown that sickle cell disease is milder in people whose red blood cells carry a fetal form of hemoglobin. The healthy fetal hemoglobin compensates for the mutated “adult” hemoglobin that makes red blood cells stiffen and assume the classic “sickle” shape. Normally, fetal hemoglobin production tails off after birth, shut ... Read More about BCL11A-based gene therapy for sickle cell disease passes key preclinical test
Tagged: blood, gene therapy, sickle cell disease
Wine used to toast CGD gene therapy trial linked to decades-long scientific journey
When Brenden Whittaker of Columbus, Ohio, the first patient treated with gene therapy for chronic granulomatous disease (CGD), showed successful engraftment last winter, the gene therapy team lifted glasses for a celebratory toast. The wine they sipped was no ordinary wine. The 2012 Bordeaux blend came from an award-winning California vineyard owned and operated by Robert ... Read More about Wine used to toast CGD gene therapy trial linked to decades-long scientific journey
Tagged: gene therapy, hematology
Forty years waiting for a cure: ALD gene therapy trial shows early promise
A small piece of notepaper, folded twice, sits tucked in a slot of the secretary desk in the living room. Every so often, I pull it out, read it, then reread. Addressed to my mom, the paper has a question and two boxes, one “yes” and one “no,” written with the careful precision of a ... Read More about Forty years waiting for a cure: ALD gene therapy trial shows early promise
Tagged: adrenoleukodystrophy, gene therapy, stem cells
Gene therapy restores hearing in deaf mice
More than 70 different genes are known to cause deafness when mutated. Jeffrey Holt, PhD, envisions a day when patients with hearing loss have their genome sequenced and their hearing restored by gene therapy. A proof-of-principle study published today by the journal Science Translational Medicine takes a clear step in that direction, restoring hearing in deaf mice. “Our gene ... Read More about Gene therapy restores hearing in deaf mice
Tagged: gene therapy, hearing
Gene therapy gets in the ring with another disease
Seeing that his mother, Kadriye, wasn’t looking, Emir Seyrek got an impish grin on his face, the kind only a two-year-old can have. He quietly dumped his bowl of dry cereal out on his bed and, with another quick look towards his mother, proceeded to pulverize the flakes to dust with his toy truck. The ... Read More about Gene therapy gets in the ring with another disease
Tagged: gene therapy, immune disorders
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
Tagged: gene therapy, rare disease
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
Tagged: gene therapy, scid
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
Tagged: gene therapy, hearing
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
Tagged: cancer, clinical trials, gene therapy, immune disorders