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When Jesse was 18, he decided to volunteer for a gene therapy trial at the University of Pennsylvania. He had a severe adverse reaction and died four days later. Jesse’s death has become an iconic story of tragedy and misconduct, of greed and ambition. Many accounts of his death also lament that it set the field of gene therapy back ten years.
May 7, 2015 · Center for Genetics and Society, Human genetic modification (or “gene editing”) can be used in two very different ways. Somatic genome editing changes the genes in a patient’s cells to treat a medical condition. A few gene therapies are approaching clinical use but remain extraordinarily expensive. By contrast, heritable genome editing ...
But physicians say gene therapy is likely to yield even better results, providing normal or near-normal hearing ability to thousands of people born with rare types of deafness. More than a decade after the first successful gene therapy was tested at CHOP, the technology has come of age, enabling doctors to treat conditions from...
Gene Therapy: Changing genomes to treat disease. There are two distinct ways gene editing might be used in humans. Gene therapy, or somatic gene editing, changes the DNA in cells of an adult or child to treat disease, or even to try to enhance that person in some way. The changes made in these somatic (or body) cells would be permanent but ...
Apr 29, 2024 · The Institute for Clinical and Economic Review (ICER), a health-economics think tank in Boston, analyzed the treatment last year. They concluded that it might be cost-effective at a price “ between $2.3m and $3.9m. ” That range is of course high and wide, but the company chose a price point $350,000 to almost $2m above it.
Doctors have recently looked to gene therapy as an experimental technique to treat diseases including cancer, ALS and Alzheimer’s disease. But disorders such as ADHD and autism have not been part of that discussion. Wells and his colleagues found that genetically modified mice missing the “Ptchd1” gene — suspected to be absent in some ...
Aug 22, 2024 · Opinions about Heritable Human Genome Editing. The main focus in this section is on heritable genetic modification (also called human germline modification or reproductive gene editing), though it also includes some results concerning somatic gene editing (gene therapy) and some other issues such as sex selection.
Jan 23, 2024 · Last year, after moving to Spain, his family took him to a hearing specialist, who made a surprising suggestion: Aissam might be eligible for a clinical trial using gene therapy. On Oct. 4, Aissam was treated at the Children’s Hospital of Philadelphia, becoming the first person to get gene therapy in the United States for congenital deafness.
Aug 16, 2022 · The redemption of James Wilson, gene therapy pioneer. In recent years, Wilson has warned about the potential dangers involved in gene therapy, particularly the use of high doses of adeno-associated virus (AAV) vectors to deliver DNA. Indeed, in 2018 he resigned as a scientific advisor to Solid Biosciences because of concerns about the safety of ...
Mar 2, 2017 · A recent study in mice, however, published in the journal Molecular Therapy, describes a new method for delivering genes to the sensory hair cells of the inner ear as a potential treatment for deafness. This research describes a new type of viral vector, exo-AAV1, which is more efficient than AAV1 and which may be an effective viral vector for delivering therapeutic genes to treat hereditary deafness by gene therapy.
