Terapia Génica para Encefalopatías Congénitas
Great Ormond Street Hospital for Children NHS Foundation Trust
Londres, Reino UnidoPublications in collaboration with researchers from Great Ormond Street Hospital for Children NHS Foundation Trust (1)
2022
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Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors
Molecular Therapy - Methods and Clinical Development, Vol. 27, pp. 96-108