Editas Medicine says its ex vivo gene-editing therapy for hemoglobinopathies has best-in-class potential, but the company plans to out-license or partner that program. Editas is shifting focusing ...

CRISPR Therapeutics CRSP and Intellia Therapeutics NTLA are leading developers of therapies that utilize the Nobel ...

CRISPR Therapeutics CRSP is currently the only company in the world to have secured approval for a CRISPR-based gene therapy developed in partnership with Vertex Pharmaceuticals. Following the ...

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

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 ...

With an ex vivo therapy, a patient's cells must be harvested from their bone marrow or blood, then transported to a lab and re-engineered, using CRISPR/Cas9, to be capable of attacking e.g ...

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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 ...

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.

What’s driving the quest for in vivo gene therapy rather than ex vivo? All gene therapy approaches currently used in the clinic for genetic diseases are ex vivo, meaning we have to take the patient’s ...

Following the landmark success with Casgevy, an ex vivo therapy, the company is now focusing on in vivo candidates. Unlike ex vivo therapies, where cells are removed, modified and then inserted ...