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HTT Aggregate Transfer in Stem Cell-based Therapy

The transplantation of gene-edited stem cells represents a promising strategy for the treatment of neurodegenerative diseases. Huntington's disease (HD) is one such neurodegenerative diseases in which this strategy has been studied; however, the transfer of mutant huntingtin (HTT) protein aggregates from HD cells to transplanted stem cells in the host brain by a “prion‐like” mechanism represents a recently-identified issue and brings into a question to what extent stem cell/gene‐corrected cell transplants are viable for the treatment of HD. For more on this fascinating topic, see the recent STEM CELLS Translational Medicine Concise Review article from the laboratory of Julien Rossignol (Central Michigan University, Mount Pleasant, Michigan USA).