Researchers use targeted lipid nanoparticles loaded with mRNA to reprogram T cells to fight autoimmune diseases ...

Gene therapy using lentiviral vectors has already led to major clinical successes when applied ex vivo - meaning that patients' stem cells are genetically modified in the lab and reinfused after ...

The YOLT-203 in-vivo gene editing therapy was developed to treat patients living with primary hyperoxaluria type 1 (PH1), a ...

HealthDay News — Drugmaker Eli Lilly plans to buy Verve Therapeutics, a gene-editing startup, for about $1 billion upfront. The deal gives Lilly a potential new treatment for heart disease, The Wall ...

One area where gene therapy is making significant progress is in treating blood disorders. It offers hope to people with sickle cell disease or hemophilia. The research paper "In vivo ...

Isaralgagene civaparvovec is a “potential best-in-class gene therapy for Fabry disease,” according to analysts at H.C.

CRISPR "Is" The Therapy Vs CRISPR "Creates" The Therapy - Intellia's In Vivo Promise Intellia defines in vivo gene therapy using the phrase "CRISPR is the therapy", and ex vivo gene therapy as ...

All gene therapy approaches currently used in the clinic for genetic diseases are ex vivo, meaning we have to take the patient’s cells out of their body to modify them. ... For in vivo gene therapy to ...

A team of scientists from the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget ... vectors directly into the bloodstream (in vivo). The authors found that in newborn mice—and up ...

Editas Medicine is pushing its in vivo gene therapy strategy to the top of its agenda, boasting proof-of-concept data and signing a $238 million biobucks pact at the same time as it searches for a ...

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established. At present, the FDA has ...