Gene therapy pioneer Jim Wilson and researcher Kiran Musunuru are using contrasting approaches to overcome “irrational ...
The field of gene editing recently got a major boost from Massachusetts-based Beam Therapeutics, and the company’s Durham ...
The arc of biotechnology is one of breakthroughs and struggles, writes Tim Hunt, CEO of the Alliance for Regenerative ...
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
Scientists in the UK have successfully used gene therapy to restore some vision to legally blind children with an inherited retinal condition. All 11 children in the clinical trial saw ...
Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...
Scientists compared five families and multiple generations to find a gene variant that prevents COPA Syndrome and opens the door to a new gene therapy for the condition. For more than 15 years ...
A one-time injection of a novel gene therapy was able to save mice and a newborn calf from succumbing to maple syrup urine disease (MSUD), suggesting the approach could one day be used to treat ...
A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored ...
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