Study demonstrates successful gene therapy for maple syrup urine disease
A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup ...
Tag: Gene Therapy
Gene therapy breakthrough offers hope for severe Dravet syndrome cases
Researchers successfully use an adeno-associated viral vector to restore brain function in a mouse model of SCN1B-linked DEE52, reducing seizures ...
Gene therapy shows promise in treating SCN1B-linked epileptic encephalopathies
Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of symptoms in children, ...
Targeted gene therapy shuts down cancer cells' energy centers
Researchers are shining a light on cancer cells' energy centers – literally – to damage these power sources and trigger ...
Cellular Origins, Fresenius Kabi sign development agreement for scalable automation of cell and gene therapy manufacturing
Cellular Origins and Fresenius Kabi, an operating company of Fresenius, announce today they have signed a development agreement that leverages ...
Machine learning revolutionizes gene delivery
Gene therapy could potentially cure genetic diseases but it remains a challenge to package and deliver new genes to specific ...
Scientists explore early life factors contributing to SMA
Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can ...
Understanding Global Harmonization of Regulatory CMC within Veterinary Medicine with Tammy Newbold
In this interview, News-Med talks to Tammy Newbold about the Global Harmonization of Regulatory CMC within Veterinary Medicine. Can you ...
Gene editing silences mutant microRNA, restores hearing in adult mice
Researchers have used gene editing to restore hearing in adult mice with a type of inherited hearing loss. They showed ...
New gene editing technique offers hope for millions
A team led by Mass Eye and Ear researchers has demonstrated for the first time a successful restoration of hearing ...