CHOP Breakthrough in Regenerative Therapy Research: Ribosome Assembly Essential for Stem Cell Generation

CHOP Breakthrough in Regenerative Therapy Research Ribosome Assembly Essential for Stem Cell Generation

Thanks to recent research findings out of Children’s Hospital of Philadelphia (CHOP), a team has identified the genes responsible for hematopoietic stem cell (HSC) regeneration via the assembly of the ribosome, the protein factories in cells that translate mRNA sequences into amino acid sequences. The findings, which were published in Cell Stem Cell, highlight the importance of proper ribosome assembly in stem cell regeneration and identify possible targets for future therapies for ribosomopathies, childhood disorders that lead to bone marrow failure (BMF).

The study title “HectD1 Controls Hematopoietic Stem Cell Regeneration by Coordinating Ribosome Assembly and Protein Synthesis," Cell Stem Cell, online March 11, 2021, DOI: 10.1016/j.stem.2021.02.008.

What are the relevance of these CHOP researcher findings?

As senior author and Professor of Pediatric Hematology at CHOP, Wei Tong, PhD, notes, “Although previous research showed that mutations that affect ribosome assembly are associated with human diseases that involve bone marrow dysfunction, how ribosome assembly is regulated in hematopoietic stem cells and how it contributes to disease was poorly unders...

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