Using CRISPR to reverse retinitis pigmentosa and restore visual function
Using the gene-editing tool CRISPR/Cas9, researchers at University of California San Diego School of Medicine and Shiley Eye Institute at UC San Diego Health, with colleagues in China, have...
View ArticleNew studies refocus attention on the genotoxicity of AAV vectors in gene therapy
A growing number of preclinical studies in mice suggests that therapeutic gene delivery using recombinant adeno-associated viral vectors (rAAVs) can cause insertional mutagenesis and increase the risk...
View ArticleWhat causes gene transfer to trigger T cell activation and exhaustion?
Researchers are beginning to gain a clearer understanding of how the immune system responds, in both a reactive and tolerant way, to gene therapy delivered using what has become the preferred gene...
View ArticlePulling the plug on the first gene therapy drug
2017 is supposed to be the year that FDA finally approves a gene therapy. But last week, the company behind the first approved gene therapy in Europe, uniQure.com, announced that it won't "pursue the...
View ArticleWill short-term and long-term treatments for single-gene diseases survive?
Two weeks and several political disasters ago, the House of Representatives passed the American Health Care Act of 2017, and soon lists of "pre-existing conditions" festooned news feeds. We all ticked...
View ArticleBrain microenvironment makes HER2-positive breast cancer metastases resistant...
While target therapies directed toward genetic mutations that drive a tumor's growth have significantly improved the outlook for many patients, they have not been as successful in controlling brain...
View ArticleGene therapy could 'turn off' severe allergies
A single treatment giving life-long protection from severe allergies such as asthma could be made possible by immunology research at The University of Queensland.
View ArticleSafety of gene transfer to treat heart failure supports further clinical...
Based on the encouraging safety data that has emerged from multiple clinical trials that used different gene transfer approaches to improve heart function in patients suffering from heart failure with...
View ArticleGene therapy leads to long-term benefits in dog model of devastating...
Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy—a type of muscular dystrophy that affects the skeletal...
View ArticlePrednisone may improve effectiveness of AAV-based gene therapy by reducing...
A new study of gene transfer using adeno-associated virus (AAV)-based gene delivery into skeletal muscle of rhesus macaques showed that oral prednisone reduced immune responses to AAV that can weaken...
View ArticleAltered virus may expand patient recruitment in human gene therapy trials
For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment. Now, University of Florida...
View ArticleNoninvasive intranasal method shows prevention of neurologic effect from...
Researchers have successfully used a noninvasive intranasal approach to deliver the gene for the enzyme that is deficient in the inherited lysosomal storage disease mucopolysaccharidosis type 1 (MPS 1)...
View ArticleResearchers describe novel reporter proteins for long term expression of...
A new study showed that the expression levels of a novel secreted reporter protein delivered to an immunosuppressd large animal model could be detected for several months after infusion into the liver,...
View ArticleBreathing in a new gene therapy to treat pulmonary hypertension
Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension (PH), a form of high blood pressure in blood vessels...
View ArticleNovel leukemia treatment could be first US gene therapy (Update)
A treatment for a common childhood blood cancer could become the first gene therapy available in the U.S.
View ArticleNew data network for Huntington's disease research
Huntington's disease is an hereditary disorder of the nervous system caused by a faulty gene on chromosome four. The faulty gene leads to cell death in neurons in the brain resulting in gradual...
View ArticlePromising therapy for fatal genetic diseases in children nears human trials
Researchers at University of Massachusetts Medical School and Auburn University College of Veterinary Medicine are nearing human clinical trials on a genetic therapy for two rare neurological diseases...
View ArticleWhat makes cancer gene therapy so groundbreaking?
On July 12, a Food and Drug Administration panel unanimously recommended approval for the first-ever gene therapy treatment for cancer. The treatment, known as CTL019, is a T-cell therapy developed by...
View ArticleMicrodystrophin restores muscle strength in Duchenne muscular dystrophy
Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR) and the Royal Holloway University of London demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne...
View ArticleGene therapy to correct surfactant protein B deficiency in newborns
An article published in Experimental Biology and Medicine (Volume 242, Issue 13, July, 2017) reports that gene therapy may be used to as an intermediate therapy for newborns with surfactant protein...
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