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Welcome to Gene Therapy Net
Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.
Revolutionary gene therapy shows promise in treating autism
Posted on: 6 December 2023, source: Earth.com
Chinese scientists have achieved a major advancement in gene therapy for autism, as reported in the journal Nature Neuroscience. This innovative therapy, developed by a research team in Shanghai, has been successfully tested on mice. The research marks a significant step in the treatment of autism spectrum disorder (ASD) through genetic base editing within the brain.
Chinese scientists have achieved a major advancement in gene therapy for autism, as reported in the journal Nature Neuroscience. This innovative therapy, developed by a research team in Shanghai, has been successfully tested on mice. The research marks a significant step in the treatment of autism spectrum disorder (ASD) through genetic base editing within the brain.
Cell & Gene Therapy Space Gears Up for First CRISPR Approval
Posted on: 13 November 2023, source: Biospace
Halloween brings to mind pumpkins and trick or treat, but for the 100,000 Americans suffering from sickle cell disease, this past October 31 had a different meaning when an FDA advisory panel deemed the first potentially curative SCD therapy safe enough for clinical use, bolstering hopes of an FDA approval in December. It would also be the first-ever approval for CRISPR, the genetic modification technique that won its discoverers the Nobel Prize in 2020 for their 2012 breakthrough.
Halloween brings to mind pumpkins and trick or treat, but for the 100,000 Americans suffering from sickle cell disease, this past October 31 had a different meaning when an FDA advisory panel deemed the first potentially curative SCD therapy safe enough for clinical use, bolstering hopes of an FDA approval in December. It would also be the first-ever approval for CRISPR, the genetic modification technique that won its discoverers the Nobel Prize in 2020 for their 2012 breakthrough.
Gene therapy restores hearing in children with rare form of deafness
Posted on: 28 October 2023, source: Science
Several deaf children can hear after receiving gene therapy—a first for the approach—a team at Fudan University reported today at a meeting in Belgium. The children were born deaf because they inherited two defective copies of the gene for a protein called otoferlin that helps the inner ear’s hair cells transmit sound to the brain. In an attempt to restore this function, researchers injected harmless viruses carrying DNA for a working copy of the otoferlin gene into the children’s ears. Four of five patients treated now have some hearing, the MIT Technology Review reports.
Several deaf children can hear after receiving gene therapy—a first for the approach—a team at Fudan University reported today at a meeting in Belgium. The children were born deaf because they inherited two defective copies of the gene for a protein called otoferlin that helps the inner ear’s hair cells transmit sound to the brain. In an attempt to restore this function, researchers injected harmless viruses carrying DNA for a working copy of the otoferlin gene into the children’s ears. Four of five patients treated now have some hearing, the MIT Technology Review reports.
UK biotech AlveoGene launches with plans for inhaled gene therapy
Posted on: 16 September 2023, source: BiopharmaDive
The company aims to capitalize on research by the Respiratory Gene Therapy Consortium, and is working first on a treatment of alpha-1 antitrypsin deficiency. The unusual approach could be a way to more reliably deliver gene therapies directly to the epithelial cells of the lungs. The company said preclinical research suggests its technology — dubbed InGenuiTy — could work with high efficiency and long-lasting effects. AlveoGene aims to bring its first candidate, a treatment for patients with alpha-1 antitrypsin deficiency or AATD, into clinical testing over the next two to three years. The company said it will also look to combine its platform with other technologies to target additional diseases such as lung surfactant deficiencies and idiopathic pulmonary fibrosis.
The company aims to capitalize on research by the Respiratory Gene Therapy Consortium, and is working first on a treatment of alpha-1 antitrypsin deficiency. The unusual approach could be a way to more reliably deliver gene therapies directly to the epithelial cells of the lungs. The company said preclinical research suggests its technology — dubbed InGenuiTy — could work with high efficiency and long-lasting effects. AlveoGene aims to bring its first candidate, a treatment for patients with alpha-1 antitrypsin deficiency or AATD, into clinical testing over the next two to three years. The company said it will also look to combine its platform with other technologies to target additional diseases such as lung surfactant deficiencies and idiopathic pulmonary fibrosis.
Gene Therapy Targets Chronic Pain
Posted on: 28 August 2023, source: Medgadget
Scientists at New York University have developed a gene therapy for chronic pain. The technology works by targeting the NaV1.7 sodium ion channel present on neurons, which is an important component of the pain response. The researchers encoded a version of a peptide that allows a modulatory protein, called CRMP2, to bind to NaV1.7 sodium ion channels and modulate their activity. Treating neurons so that they now express this peptide interfered with the ability of CRMP2 to affect the sodium channel, reducing the transmission of pain. As chronic pain affects a large number of patients, new treatments such as this could be set to make a real difference in many lives.
Scientists at New York University have developed a gene therapy for chronic pain. The technology works by targeting the NaV1.7 sodium ion channel present on neurons, which is an important component of the pain response. The researchers encoded a version of a peptide that allows a modulatory protein, called CRMP2, to bind to NaV1.7 sodium ion channels and modulate their activity. Treating neurons so that they now express this peptide interfered with the ability of CRMP2 to affect the sodium channel, reducing the transmission of pain. As chronic pain affects a large number of patients, new treatments such as this could be set to make a real difference in many lives.
Gene therapy eyedrops restored a boy's sight. Similar treatments could help millions
Posted on: 26 July 2023, source: Albuquerque Journal
Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye. Antonio, who's been legally blind for much of his 14 years, can see again. The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. That gave Sabater an idea: What if it could be adapted for Antonio's eyes?
Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye. Antonio, who's been legally blind for much of his 14 years, can see again. The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. That gave Sabater an idea: What if it could be adapted for Antonio's eyes?
Gene therapy approved by FDA for certain boys with rare genetic condition
Posted on: 10 July 2023, source: NBC Chicago
A new gene therapy treatment gained FDA approval last month to treat boys ages four and five with Duchenne muscular dystrophy, a rare and deadly genetic disorder that leads to muscle degeneration. Connor Stoll from Chicago was the first patient in the nation to receive the infusion as part of a clinical trial at Nationwide Children’s Hospital in January 2018.
A new gene therapy treatment gained FDA approval last month to treat boys ages four and five with Duchenne muscular dystrophy, a rare and deadly genetic disorder that leads to muscle degeneration. Connor Stoll from Chicago was the first patient in the nation to receive the infusion as part of a clinical trial at Nationwide Children’s Hospital in January 2018.
Gene Therapy News
- Gene Therapy Breakthrough: Lyfgenia for Sickle Cell Disease Treatment - Medriva
Sun, 10 Dec 2023 23:38:5 GMT - Sickle Cell Gene Therapy 'Truly Transformative' - Medscape
Sun, 10 Dec 2023 23:32:0 GMT - Gene Therapy for Sickle Cell Disease: Lovotibeglogene Autotemcel Treatment - Medriva
Sun, 10 Dec 2023 23:29:0 GMT - Long-Term Data Presented at ASH Support beti-cel as a Potentially Curative Gene Therapy for β-Thalassemia Patients ... - Business Wire
Sun, 10 Dec 2023 18:02:0 GMT - FDA Approves bluebird bio's Lovo-Cel Gene Therapy for Sickle Cell Disease - AJMC.com Managed Markets Network
Sun, 10 Dec 2023 14:28:4 GMT
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