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Ensuring a future for gene therapy for rare diseases

Alessandro Aiuti, Francesca Pasinelli, Luigi Naldini

2022Nature Medicine56 citationsDOI

Topics & Concepts

Genetic enhancementIntensive care medicineDiseaseMedicineHematopoietic stem cellHaematopoiesisStem cellGeneInternal medicineBiologyGeneticsImmunodeficiency and Autoimmune DisordersNeurogenetic and Muscular Disorders ResearchVirus-based gene therapy research
Ensuring a future for gene therapy for rare diseases | Litcius