Seeing hope: FDA panel considers gene therapy for blindness (Update)
A girl saw her mother's face for the first time. A boy tore through the aisles of Target, marveling at toys he never knew existed. A teen walked onto a stage and watched the stunned expressions of...
View ArticleFDA panel mulls gene therapy for kids with rare eye disease (Update)
(HealthDay)—A U.S. Food and Drug Administration advisory panel was poised on Thursday to recommend approval for a gene therapy that could grant the gift of sight to young people with a rare type of...
View ArticleGerman research advances in cancer and blood disorders reported in human gene...
Virotherapy capable of destroying tumor cells and activating anti-tumor immune reactions, and the use of engineered hematopoietic stem cells (HSCs) to deliver replacement genes that have the potential...
View ArticleUS regulators approve 2nd gene therapy for blood cancer
U.S. regulators on Wednesday approved a second gene therapy for a blood cancer, a one-time, custom-made treatment for aggressive lymphoma in adults.
View ArticleResearch advance may prevent a form of hereditary hearing loss
A research advance co-led by Case Western Reserve University School of Medicine's Kumar Alagramam, PhD, may stop the progression of hearing loss and lead to significant preservation of hearing in...
View ArticleGene therapy protects against age-related cognitive and memory deficits
Researchers from the Institute of Neurosciences at the Universitat Autònoma de Barcelona (INc-UAB) and the Vall d"Hebron Research Institute (VHIR) are the first to demonstrate that regulation of the...
View ArticleSubset of stem cells identified as source for all cells in blood and immune...
Researchers at Fred Hutchinson Cancer Research Center have identified a specific subset of adult blood stem cells that is exclusively responsible for repopulating the entire blood and immune system...
View ArticlePhase 1 study shows encouraging data for gene replacement therapy for SMA type I
A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical trial, according to a study published...
View ArticleNew gene therapy targets brain disorders
UNSW researchers have tailored gene therapy for a rare genetic disorder called Canavan disease, and say it could eventually be adapted to treat other inherited disorders affecting the brain.
View ArticleGenetic treatment for blindness may soon be reality
Patients who had lost their sight to an inherited retinal disease could see well enough to navigate a maze after being treated with a new gene therapy, according to research presented today at AAO...
View ArticleUS scientists try first gene editing in the body
Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person's DNA to try to cure a disease.
View ArticleNew gene therapy transplantation technique could improve treatment of...
A therapeutic technique to transplant blood-forming (hematopoietic) stem cells directly into the brain could herald a revolution in our approach to treating central nervous system diseases and...
View ArticleOne-dose gene therapy produces clotting factor, safely stops bleeding in...
A team of gene therapy researchers has reported positive results in a phase 1/2 clinical trial for the inherited bleeding disorder hemophilia B. A single intravenous infusion of a novel bioengineered...
View ArticleGene therapy shows promise against blood-clotting disease
Gene therapy has freed 10 men from nearly all symptoms of hemophilia for a year so far, in a study that fuels hopes that a one-time treatment can give long-lasting help and perhaps even cure the blood...
View ArticleInflammatory factors linked to inhibition of factor VIII gene therapy in...
As a gene therapy cure for hemophilia A moves closer to reality, a new study sheds light on a challenging complication in which a host autoimmune response inhibits the production of normal clotting...
View ArticleGene therapy improves immunity in babies with 'bubble boy' disease
Early evidence suggests that gene therapy developed at St. Jude Children's Research Hospital will lead to broad protection for infants with the devastating immune disorder X-linked severe combined...
View ArticleGroundbreaking gene therapy trial set to cure hemophilia
A 'cure' for haemophilia is one step closer, following results published in the New England Journal of Medcine of a groundbreaking gene therapy trial led by the NHS in London.
View ArticleThe story of a new skin
Decades of scientific research into areas including plant genetics and data science helped doctors successfully carry out an experimental therapy to create a new skin for a seven-year-old boy suffering...
View ArticleEarly diagnosis can save babies' lives: A guide to severe combined...
A new review provides guidance on a deadly, but rare, disease that is potentially curable if identified early. Severe combined immunodeficiency disease (SCID), known as the "bubble boy disease" in the...
View ArticleGene therapy for rare form of blindness wins US approval
U.S. health officials on Tuesday approved the nation's first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness. It marks another major...
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