Would expression of GroEL and GroES in erythrocytes be a potentially effective therapeutic strategy for sickle cell disease? Why or why not?

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    $\begingroup$ Apart from the answers from WYSIWYG I would add that the formation of filaments of Hb in sickle cells isn't really misfolding or misassembly, rather it is due to a new assembly pathway which results from the Glu>Val mutation. GroEL helps a protein to avoid kinetic traps en route to folding, filament formation is an extra process tacked on at the end of normal folding. $\endgroup$
    – Alan Boyd
    Commented Mar 15, 2014 at 7:24

1 Answer 1

  1. GroEL and GroES are multiprotein chaperonin complexes: Not so easy to ectopically express
  2. Erythrocytes do not have nucleus: you cannot stably express anything in mature erythrocytes.
  3. If your aim is to do a stable gene expression (which would require genomic integration), the better way is to do a gene therapy: culture haematopoetic stem cells, Correct the mutation by homologous recombination. Put the cells back into the bone marrow.

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