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 ...

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 amount of blood the ... Scientists Identify a ...

Researchers at Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine ...

The science has proven that gene therapies are effective treatments, and they have given hope to patients and their families ...

Intrathecal onasemnogene abeparvovec (OAV101IT) is an investigational, one-time gene therapy for patients with spinal muscular atrophy (SMA). The STEER study is a phase III randomized, double ...

Each gene tells a cell to make a specific molecule, but this isn't performed by the DNA itself. Instead, the information is relayed to cellular machinery by a molecule called messenger RNA.

New work by Columbia researchers has turned a textbook principle of genetics on its head and revealed why some people who ...

Novartis NVS announced positive top-line results from the late-stage STEER study on experimental candidate intrathecal ...

A team at the University of Calgary is making scientific advances in understanding plant adaptation, which could ultimately be used to find ways to make agricultural crops more resilient to heat ...

The company made its debut last year with a $51 million Series A and a licensing agreement with Astellas for a gene therapy candidate (KT430) for X-linked myotubular myopathy. Shortly after that ...