Clinical applications of gene therapy for rare diseases: A review

Papaioannou, I; Owen, JS; Yáñez-Muñoz, RJ

Yáñez-Muñoz, RJ (通讯作者),Royal Holloway Univ London, Ctr Gene & Cell Therapy, Ctr Biomed Sci, Sch Life Sci & Environm,AGCTlab.org, Egham TW20 0EX, England.

INTERNATIONAL JOURNAL OF EXPERIMENTAL PATHOLOGY, 2023; 104 (4): 154

Abstract

Rare diseases collectively exact a high toll on society due to their sheer number and overall prevalence. Their heterogeneity, diversity, and nature p......

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