Glaser Juliane, Cova Giulia, Fauler Beatrix, Prada-Medina Cesar A, Stanislas Virginie, Phan Mai H Q, Schöpflin Robert, Aktas Yasmin, Franke Martin, Andrey Guillaume, Bartzoka Natalia, Paliou Christina, Laupert Verena, Chan Wing-Lee, Wittler Lars, Mielke Thorsten, Mundlos Stefan
RG Development & Disease, Max Planck Institute for Molecular Genetics, Berlin, Germany.
Department of Pathology, New York University School of Medicine, Langone Health Medical Center, New York, NY, USA.
Nat Genet. 2025 Jul 9. doi: 10.1038/s41588-025-02248-5.
Transposable elements (TEs) are scattered across mammalian genomes. Silencing of TEs prevents harmful effects caused by either global activation leading to genome instability or insertional mutations disturbing gene transcription. However, whether the activation of a TE can cause disease without directly affecting gene expression is largely unknown. Here we show that a TE insertion can adopt nearby regulatory activity, resulting in the production of cell-type-specific viral-like particles (VLPs) that affect embryo formation. Failure to silence an LTR retrotransposon inserted upstream of the Fgf8 gene results in their co-expression during mouse development. VLP assembly in the Fgf8-expressing cells of the developing limb triggers apoptotic cell death, resulting in a limb malformation resembling human ectrodactyly. The phenotype can be rescued by mutating the retrotransposon coding sequence, thus preventing its full endogenous retroviral cycle. Our findings illustrate that TE insertions can be incorporated into the local genomic regulatory landscape and that VLP production in post-implantation embryos can cause developmental defects.
转座元件(TEs)散布于哺乳动物基因组中。TEs的沉默可防止因全局激活导致基因组不稳定或因插入突变干扰基因转录而产生的有害影响。然而,TE的激活是否能在不直接影响基因表达的情况下引发疾病,在很大程度上尚不清楚。在此,我们表明TE插入可采用附近的调控活性,导致产生影响胚胎形成的细胞类型特异性病毒样颗粒(VLPs)。未能沉默插入Fgf8基因上游的LTR逆转录转座子会导致它们在小鼠发育过程中共同表达。发育中的肢体中表达Fgf8的细胞中的VLP组装引发凋亡性细胞死亡,导致类似于人类缺指(趾)畸形的肢体畸形。通过突变逆转录转座子编码序列可挽救该表型,从而阻止其完整的内源性逆转录病毒循环。我们的研究结果表明,TE插入可被纳入局部基因组调控格局,并且植入后胚胎中的VLP产生可导致发育缺陷。