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

Human mutations in PQBP1, a molecule involved in transcription and splicing, result in a reduced but architecturally normal brain. Examination of a conditional Pqbp1-knockout (cKO) mouse with microcephaly failed to reveal either abnormal centrosomes or mitotic spindles, increased neurogenesis from t...

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Detalhes bibliográficos
Publicado no:Mol Psychiatry
Main Authors: Ito, H, Shiwaku, H, Yoshida, C, Homma, H, Luo, H, Chen, X, Fujita, K, Musante, L, Fischer, U, Frints, S G M, Romano, C, Ikeuchi, Y, Shimamura, T, Imoto, S, Miyano, S, Muramatsu, S-i, Kawauchi, T, Hoshino, M, Sudol, M, Arumughan, A, Wanker, E E, Rich, T, Schwartz, C, Matsuzaki, F, Bonni, A, Kalscheuer, V M, Okazawa, H
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2015
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4378255/
https://ncbi.nlm.nih.gov/pubmed/25070536
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mp.2014.69
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