Promising results with new gene therapy approach for treating inherited...
A new gene therapy approach designed to replace the enzyme that is deficient in patients with the inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the...
View ArticleStudy verifies human gene therapy in model of rare metabolic disorder
Researchers are closer to finding a better way to treat children with a rare metabolic disorder called MPS I. It is caused by a deficiency of the key enzyme IDUA needed to break down complex sugars in...
View ArticleHow immune response differs for natural AAV infection compared to AAV vector...
A new, long-term study examined the antibody response to natural infection with adeno-associated virus (AAV) in chimpanzees for the purpose of characterizing the broad-based immune responses that could...
View ArticleThree gene therapy trials report good news
"When you hear hoofbeats, think horses, not zebras," beginning med students learn. Ultrarare diseases are more like unicorns.
View ArticleConquering the lysosomal storage disorders with genetic engineering
(Medical Xpress)—The motivation by fear, anger, and love to preserve self, kin, and others perches near the pinnacle of our existence. Triumph in the face of a seemingly insurmountable medical...
View ArticleOne-time treatment rescues lethal metabolic liver disease in mice
To treat the liver disorder tyrosinaemia type I, one of the most severe forms of the disease, doctors typically use drug therapy. However the treatment is lifelong and a residual risk of liver cancer...
View ArticleBCL11A-based gene therapy for sickle cell disease passes key preclinical test
A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from...
View ArticleSurgeons perform world's first operation inside the eye using a robot
Robert MacLaren, Professor of Ophthalmology. assisted by Dr Thomas Edwards, Nuffield Medical Fellow, used the remotely controlled robot to lift a membrane 100th of a millimetre thick from the retina at...
View ArticleGene therapy technique may help prevent cancer metastasis
The spread of malignant cells around the body, known as metastasis, is the leading cause of mortality in women with breast cancer.
View ArticleGenetic 'switch' identified as potential target for Alzheimer's disease
A team at the MRC Clinical Sciences Centre (CSC), based at Imperial College London, has found an important part of the machinery that switches on a gene known to protect against Alzheimer's Disease.
View ArticleTwo gene therapy vectors partially correct lung disease in a pig model of...
Two new studies from the University of Iowa suggest that gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis (CF).
View ArticleAdvances in stem cell therapy to treat neurogenetic diseases
Transplantation of therapeutic stem cells directly into the central nervous system (CNS) is a promising new approach to treating the neurological effects of lysosomal storage diseases (LSD), a group of...
View ArticleGene editing of blood stem cells can correct disease-causing mutations
Recent advances in gene editing technology, which allows for targeted repair of disease-causing mutations, can be applied to hematopoietic stem cells with the potential to cure a variety of hereditary...
View ArticleLong-term study shows less frequent and severe pancreatitis following gene...
Up to 6 years after receiving a single treatment with the gene therapy product lipoprotein lipase (LPL), patients with the debilitating genetic disease LPL deficiency (LPLD) had about 50% fewer...
View ArticleSafety data support human testing of hematopoietic stem cell gene therapy for...
Extensive biosafety studies of hematopoietic stem cell (HSC) gene therapy, intended to replace a protein that patients with the inherited disease mucopolysaccaridosis I (MPS I) cannot produce, support...
View ArticleAlzheimer's disease could be treated with gene therapy, animal study suggests
Researchers have prevented the development of Alzheimer's disease in mice by using a virus to deliver a specific gene into the brain.
View ArticleScientists develop a new method to measure radiation dose in cancer patients
Lawrence Livermore National Laboratory (LLNL) scientists and collaborators have developed a new technique to measure radiation dose levels using gene expression analysis of whole blood from cancer...
View ArticleIn crafting new treatments for hemophilia, a 'less is more' approach
Hematology researchers have found that blocking the role of a common protein may offer unexpected benefits for patients with the inherited bleeding disorder hemophilia A. The finding offers potential...
View ArticleThe slow climb from innovation to cure—treating anaemia with gene editing
The ability to precisely edit DNA via CRISPR technology has emerged as the one of the most powerful advances in biology. A new paper showing repair of a genetic mutation in human blood cells represents...
View Article'Gene therapy in a box' effective, reports Nature Communications
A table-top device that enables medical staff to genetically manipulate a patient's blood to deliver potential new therapies for cancer, HIV and other diseases would eliminate the need for...
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