Posts Tagged ‘Leber’s congenital amaurosis’

Giving Sight by Therapy With Genes

By Pam Belluck © The New York Times — By the time Corey Haas was 7, the retinal disease he was born with had already stolen much of his vision. “He always clung to me or my wife,” said Corey’s father, Ethan Haas. The boy relied on a cane and adults to guide him, and, unable to see blackboard writing, sat in back with a teacher’s aide, large-type computer screen and materials in braille. Legally blind, Corey was expected eventually to lose all sight. Then, 13 months ago, after his eighth birthday, he underwent an experimental gene therapy procedure, receiving an injection in his left eye. His vision in that eye improved quickly…. READ ARTICLE

Examining Gene Therapy as Treatment for Blindness

By Ira Flatow © NPR News — Reporting in The Lancet, doctors found success in treating Leber’s congenital amaurosis, a rare type of blindness, with gene therapy. Study author Katherine High explains how injecting a gene-carrying virus into the eye has improved vision in a handful of patients…. READ ARTICLE

Gene Therapy Helps Blind Boy See

By Harry Smith © CBS — Just over a year ago, 9-year-old Corey Haas and his family feared he would go blind. But, thanks to an experimental procedure, Corey has new outlook on life — and everything in it…. READ ARTICLE

Gene therapy experiment restores sight in a few

By Phil Furey © Reuters, PHILADELPHIA — Nine-year-old Corey Haas can ride his bike alone now, thanks to an experimental gene therapy that has boosted his fading vision with a single treatment. The gene therapy helped improve worsening eyesight caused by a rare inherited disease called Leber’s congenital amaurosis, or LCA, which makes most patients blind by age 40. Twelve treated patients, including Corey, now have better vision, their doctors told a joint meeting of the American Academy of Ophthalmology and Pan-American Association of Ophthalmology in San Francisco on Saturday…. READ ARTICLE

One Shot of Gene Therapy and Children with Congenital Blindness Can Now See

PRNewswire, Philadelphia — Born with a retinal disease that made him legally blind, and would eventually leave him totally sightless, the nine-year-old boy used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides. Now, after a single injection of genes that produce light-sensitive pigments in the back of his eye, he sits in front with classmates and participates in class without extra help. In the playground, he joins his classmates in playing his first game of softball. His treatment represents the next step toward medical science’s goal of using gene therapy to cure disease. Extending a preliminary study published last year on three young adults, the full study reports successful, sustained results that showed notable improvement in children with congenital blindness. The study, conducted by researchers from the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia and from the University of Pennsylvania School of Medicine, used gene therapy to safely improve vision in five children and seven adults with a Leber’s congenital amaurosis (LCA). The greatest improvements occurred in the children, all of whom are now able to navigate a low-light obstacle course—one result that the researchers call “spectacular…. READ PRESS RELEASE

Study Shows Hope for Type of Blindness

By Ron Winslow © The Wall Street Journal — A small but provocative study showed that a form of gene therapy significantly improved the vision of patients left legally blind by a rare genetic eye disease. The benefit was especially striking among children. Researchers said the findings amount to an important advance toward medicine’s ambitious but generally unrealized dream of replacing disease-causing mutant or missing genes with normal DNA to treat and cure debilitating illnesses. A young boy shows the results of eye treatment in the blind/near-blind as having remarkable effects in restoring vision. In the study, 12 patients, including four children between ages 8 and 11 years old, underwent a surgical procedure in which a gene that makes a protein critical to vision function was injected into one eye. The second eye wasn’t treated. While normal sight wasn’t restored in any of the patients, all reported some improvement. Six gained enough vision that they may no longer meet criteria for being legally blind, researchers said, including the four children for whom substantial recovery of vision appears to have transformed their lives…. READ ARTICLE