Take the 2-minute tour ×
Biology Stack Exchange is a question and answer site for biology researchers, academics, and students. It's 100% free, no registration required.

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

share|improve this question
1  
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. –  Alan Boyd Mar 15 at 7:24

1 Answer 1

up vote 3 down vote accepted
  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.
share|improve this answer

Your Answer

 
discard

By posting your answer, you agree to the privacy policy and terms of service.

Not the answer you're looking for? Browse other questions tagged or ask your own question.