In utero gene therapy rescues microcephaly caused by Pqbp1-hypofunction in neural stem progenitor cells

H. Ito, H. Shiwaku, C. Yoshida, H. Homma, H. Luo, X. Chen, K. Fujita, L. Musante, U. Fischer, S. G.M. Frints, C. Romano, Y. Ikeuchi, T. Shimamura, S. Imoto, S. Miyano, S. I. Muramatsu, T. Kawauchi, M. Hoshino, M. Sudol, A. ArumughanE. E. Wanker, T. Rich, C. Schwartz, F. Matsuzaki, A. Bonni, V. M. Kalscheuer, H. Okazawa

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Medicine & Life Sciences