To elucidate principles operating in native biological systems and to develop novel
biotechnologies, synthetic biology aims to build and integrate synthetic gene circuits within …

Genomic safe harbors (GSHs) are sites in the genome able to accommodate the integration
of new genetic material in a manner that ensures that the newly inserted genetic elements:(i) …

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder due
to selective loss of motor neurons (MNs). Mutations in the fused in sarcoma (FUS) gene can …

The development of complex in vitro hepatic systems and artificial liver devices has been
hampered by the lack of reliable sources for relevant cell types, such as hepatic stellate cells …

TDP‐43 is the major component of pathological inclusions in most ALS patients and in up to
50% of patients with frontotemporal dementia (FTD). Heterozygous missense mutations in …

Predicting drug-induced liver injury in a preclinical setting remains challenging, as cultured
primary human hepatocytes (PHHs), pluripotent stem cell-derived hepatocyte-like cells …

Animal testing is still the most popular preclinical assessment model for liver fibrosis. To
develop efficient anti-fibrotic therapies, robust and representative in vitro models are …

The isolation or in vitro derivation of many human cell types remains challenging and
inefficient. Direct conversion of human pluripotent stem cells (hPSCs) by forced expression …

Imaging reporter genes provides longitudinal information on the biodistribution, growth, and
survival of engineered cells in vivo. A translational bottleneck to using reporter genes is the …

Precisely sensing and guiding cell-state transitions via the conditional genetic activation of
appropriate differentiation factors is challenging. Here we show that desired cell-state …