Improved Prime Editing System Makes Gene-Sized Edits in Human Cells at Therapeutic Levels June 10, 2024 — Scientists have improved a gene-editing technology that is now capable of inserting or ...

A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.

The evolution of cell and gene therapy is reshaping drug discovery, providing new hope for rare diseases and cancer through ...

CRISPR technology, driven by Cas9, redefines gene editing, facilitating targeted DNA modifications that advance genetic ...

With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery ...

Regeneron’s gene therapy, dubbed DB-OTO, is a cell-selective AAV gene therapy for children with hearing loss stemming from a ...

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...

The companies were two years into a four-year, $400 million agreement aimed at developing and marketing gene therapies ...

YolTech's gene therapy is intended as a one-time life-long cure for patients living with PH1, which can lead to kidney ...

What's more, Tas proteins are compact—a quarter of the size of Cas9 on average—making them easier to deliver, which could overcome a major obstacle to therapeutic deployment of gene editing tools.

The "AAV Vectors in Gene Therapy - Pipeline Insight, 2025" report has been added to ResearchAndMarkets.com's offering.The Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline signifies a ...

A vast search of natural diversity has led scientists at MIT’s McGovern Institute and the Broad Institute of MIT and Harvard ...