Gene and Cell Therapy – News and Features

Researchers used glioblastoma-derived organoids to predict patient responses to CAR T cell therapy with remarkable accuracy. These organoids mirrored the tumor’s reaction to treatment in real time, offering insights into efficacy and neurotoxicity.

UCSF researchers have engineered T cells to temper overactive immune responses by targeting inflammation and soothing immune activity. The technology successfully protected transplanted pancreatic islet cells in a type 1 diabetes model.

Researchers have uncovered surprising ways transcription factors – the genetic switches for genes – regulate plant development, revealing how subtle changes in a lipid-binding region can dramatically affect growth.

Researchers have built a synthetic receptor with broad potential to program cell activity, including immune response and neurological signaling.

The therapy molecule AZD7648 can promote precise DNA repair during CRISPR gene editing, but creates massive genetic changes elsewhere.

A novel CAR T-cell therapy has benefitted patients with relapsed acute lymphoblastic leukemia, showing high response rates in a Phase Ib/II trial.

Dr. Eric Kelsic, CEO and co-founder of Dyno Therapeutics, explains how AI could reduce the cost of gene therapy delivery to $0.

UCSF's clinical trial uses CRISPR-Cas9 gene editing to directly correct the genetic mutation causing sickle cell disease. By modifying patients' own blood stem cells without viral vectors, the therapy offers a safer alternative to donor transplants.

Researchers developed a gene therapy to enhance placental function by delivering IGF-1 DNA using nanoparticles. Tested in rhesus macaques, the therapy improved placental health without adverse effects.

Using adeno-associated viruses (AAVs), scientists mapped neural circuits in frogs, tracing changes during metamorphosis. This method reveals how amphibian nervous systems adapt as locomotion transitions from swimming to walking.