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diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency
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World’s First Patient Treated with Personalized base editing system !
This proof of principle paves the way for precision gene therapy administered directly in vivo. In addition to having a significant therapeutic effect, this trial will allow the collection of critical data for designing tomorrow's precision gene therapies, both in terms of efficacy and biosafety. We know that the liver, a target organ in this pathology, is a prime target for in vivo gene therapy because after systemic injection, vectors accumulate there without a specific targeting system and liver cells multiply rapidly, allowing rapid colonization of cured cells. Consequently, these results support the idea that the field of vectorology is key to harnessing the power of new genome editors such as base editing systems, prime editing systems. The design of vectors that can target the desired cell populations, without compromising efficacy and biosafety, is the next critical step to enable precision gene therapy for a large number of genetic pathologies.