Gene and Cell Therapy – News and Features

New research has illustrated how hepatitis A virus uses cellular proteins, like PDAP1, to infect liver cells, replicate and spread.

RNA therapeutics promise rapid, personalized and flexible treatments against multiple diseases. In this article, we will explore whether RNA therapeutics can realize their full potential.

A new gene editing therapy for a rare heart condition has shown promise in an early trial.

A novel CAR-T therapy targeting p95HER2 shows complete and durable tumor responses in HER2+ breast cancer preclinical models. Combining CAR-T cells with a bispecific antibody, this approach enhances immune activation while avoiding toxicities.

Researchers developed a dual-AAV gene therapy for Usher syndrome type 1F, restoring hearing and balance in mice. The approach effectively delivered the large PCDH15 gene and showed promise in retinal organoids and nonhuman primates.

A new technology that delivers two halves of a gene separately could enable gene therapy treatments of muscular dystrophies.

Researchers at UC San Francisco developed SNIPRs, sensors that activate engineered cells only in the tumor environment, offering targeted cancer treatment. When tested in mice, the SNIPR-equipped cells attacked only tumor cells.

A Stanford Medicine trial has shown success with CAR-T cell therapy in shrinking deadly childhood brain tumors. For one patient, tumor traces disappeared completely, marking a promising development in treating diffuse intrinsic pontine glioma (DIPG).

Researchers at the University of Basel have developed CAR T-cells that target glioblastoma, a notoriously aggressive brain cancer. This approach not only attacks tumor cells directly but also neutralizes the tumor’s supportive microenvironment.

UW–Madison researchers used gene therapy to prevent hereditary spastic paraplegia (HSP) in a rat model, introducing a healthy version of the Trk-fused gene to compensate for the mutated one. This prevented the onset of HSP symptoms in rats.