This research evaluates the effects of various gene therapy viral vectors on RPE cells to determine efficacy for therapeutic ...

According to Alnylam’s Tim Mooney, mivelsiran’s endurance is due to stabilizing tweaks the scientists made to the siRNA ...

A patient has died after receiving the high dose of Neurogene’s Rett syndro | A patient has died after receiving the high ...

It delivers a functional copy of the F9 gene directly to the liver cells using an adeno-associated virus (AAV) vector. Once ...

OXB to host a free webinar on accelerating development of viral vectors through automated manufacturing and analytical ...

SRD-001/2/3 employ an adeno-associated virus (AAV)-based vector system, delivered directly to cardiac ventricular muscle cells via Sardocor’s proprietary intracoronary infusion system. Furthermore, ...

Siren Biotechnology's Approach Could Transform the High-Grade Glioma Therapy Landscape "SRN-101 has the potential to address ...

Following this update, the company's stock price dropped more than 30 percent from market close Friday to $23.79 per share Monday morning.

Researchers have developed a dual-gene therapy approach to treat Usher syndrome type 1F, a rare condition causing deafness and progressive blindness.

Researchers combine gene therapy with optogenetics to curb abnormal neural activity and prevent seizure-like activity in neurons.

The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...

Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...