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🇬🇧·1d agoResearch

Beneficial bystander-enhanced cryptic splice rescue of cardiac-type Fabry GLA IVS4+919G>A by adenine base editing in patient fibroblasts

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Nature Gene Therapy

UK

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Source: Nature Gene Therapy

Scope: Research

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Beneficial bystander-enhanced cryptic splice rescue of cardiac-type Fabry GLA IVS4+919G>A by adenine base editing in patient fibroblasts | PharmaRadar360