BEYOND THE BUBBLE: 10 Years of Gene Therapy 10 minute documentary/feature created as my end of year project for my Masters course in TV Journalism at Nottingham Trent University. Made by David Sykes on... By: David Sykes … Continue reading →

Genetic Researching: Gene Therapy Bio Video. By: Ali Banach … Continue reading →

Researchers used an emerging technique to correct the gene behind a fatal immune system disorder in an infant. A new kind of gene therapy which involves editing, rather than replacing, faulty genes in sick people, is being used experimentally in patients. The latest report shows how scientists can correct a broken gene as it sits in the patients genome. How the health of the patient, a 4-month old infant, will change is yet to be reported. Genome editing technology is considered a promising new tool for curing disease. For decades, gene therapy has meant that a virus delivers a functional copy of a gene that is dysfunctional in a patient. The dysfunctional copy remains and the therapeutic version typically remains separate from the rest of the genome. The technology has drawbacks. First, by sitting outside of the genome, the activity of therapeutic gene isnt regulated properly. In some cases, the therapeutic copy is delivered by a retrovirus the plunks the new gene down near randomly in the patients genome, which risks disrupting another gene, potentially causing cells to turn cancerous. Second, some diseases, such as Huntingtons, cant be treated this way because the broken copy of the gene causes harm. … Continue reading →

AChildrens Hospital of Philadelphia spinout with the potential to be the first to win U.S. FDA approval of a gene therapy is now funded through the rest of development of its lead treatment for inherited blindness. Spark Therapeutics is in the middle of a Phase 3 clinical trial that will determine whether the gene therapy its developed can successfully restore some vision to people with rare degenerative eye diseases caused by mutations in the RPE65 gene. Theres currently no treatmentavailable for these diseases. To allow Spark to continue that study and prepare for commercialization, the company has just closed an oversubscribed $72.8 million Series B financing. Investors including Sofinnova Ventures, Brookside, Deerfield, Rock Springs Capital, T. Rowe Price, Wellington and CHOP participated. Developed at CHOPs Center for Cellular and Molecular Therapeutics, the therapy uses a neutralized virus as a vehicle to deliver a function gene to targeted cells in the eye. Once there, it enables production of a critical protein thats missing as a result of a mutation and causes vision loss. In earlier studies, the company reported that some children who were nearly blind as the result of a RPE65 mutation were able to recognize faces and walk without … Continue reading →