Manipulation of protein translation and stem cell self-renewal by CRISPR activation of rRNA transcription

Манипулирование трансляцией белка и самоподдержанием стволовых клеток с помощью активации транскрипции rRNA CRISPR
Magdalena Götz, Jovica Ninkovic, Paul A. Trainor, Emilie Alard, Faraz K. Mardakheh, Stefan H. Stricker, Anna Köferle, Stefanie M. Hauck, Markus E. Diefenbacher, Anna Danese, Maximilian Wiesbeck, Florencia Merino, Niti Chowdhury, Luisa Egert, Simon Imhof, Matilde Iraci Borgia, Akshaya Rajan, Nadine Fernandez-Novel Marx, Edina Kepesidis, Luis Miguel Cerron-Alvan, Franziska Vierl, T. Truong, Manja Thorwirth, Lorina Bilalli, Rico Schieweck
2026-07-02

47S rDNA transcriptionCRISPR activationTAPIRneural stem cell self-renewalrRNA transcription
Ribosomal RNA (rRNA) transcription rates vary during development, and their dysregulation is linked to diseases such as cancer and ribosomopathies. Owing to their high abundance and genomic redundancy, the functional significance of rRNA-levels remains unclear. Here, we developed TAPIR (Targeted Activation of Protein Translation), a CRISPR-based approach to elevate rRNA-levels by inducing 47S rDNA transcription. TAPIR increased nucleolar size and enhanced protein synthesis, even in rapidly proliferating cells. In neural stem cells, elevated translation promoted self-renewal and proliferation in vitro and in vivo. Furthermore, TAPIR enabled the modeling and partial rescue of associated disease phenotypes. Our findings revealed that rRNA-levels directly regulate translational output and that protein synthesis capacity can act as a key determinant of mammalian stem cell behavior.
1
Developed TAPIR, a CRISPR-based method to elevate 47S rDNA transcription and thereby increase rRNA levels.
2
Elevated translation via TAPIR promoted neural stem cell self-renewal and proliferation both in vitro and in vivo.
3
TAPIR enabled modeling of disease phenotypes linked to rRNA dysregulation and facilitated partial rescue of those phenotypes.
4
TAPIR-induced rRNA upregulation enlarged nucleolar size and enhanced global protein synthesis, including in rapidly proliferating cells.
5
rRNA levels directly regulate translational output, and protein synthesis capacity can determine mammalian stem cell behavior.

Cellular ribosomal RNA (rRNA) levels manipulated via CRISPR-based TAPIR to induce 47S rDNA transcription

Effects of elevated rRNA levels on protein translation capacity, nucleolar size, and stem cell self-renewal and proliferation (including in vitro and in vivo behavior and disease-phenotype modeling/rescue)

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2026-07-02
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Magdalena Götz
Jovica Ninkovic
Paul A. Trainor
Emilie Alard
Faraz K. Mardakheh
Stefan H. Stricker
Anna Köferle
Stefanie M. Hauck
Markus E. Diefenbacher
Anna Danese
Maximilian Wiesbeck
Florencia Merino
Niti Chowdhury
Luisa Egert
Simon Imhof
Matilde Iraci Borgia
Akshaya Rajan
Nadine Fernandez-Novel Marx
Edina Kepesidis
Luis Miguel Cerron-Alvan
Franziska Vierl
T. Truong
Manja Thorwirth
Lorina Bilalli
Rico Schieweck
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