re: apart from the potential CAR-T applications, SGMO's programs should be confined primarily to aut | SGMO Message Board Posts

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Msg  84615 of 85221  at  1/11/2017 2:49:06 PM  by

Watsonncrick

The following message was updated on 1/11/2017 3:52:03 PM.

re: apart from the potential CAR-T applications, SGMO's programs should be confined primarily to autologous HSCs (e.g., the BCL11a enhancer program) so #1 about improved conditioning regimens to "make room" for gene edited HSCs is worth reading

  
  
  
  
 I've posted previously about reviews that summarize "state of the art" approaches to hematopoietic stem cell (HSC) gene editing mentioning that less toxic conditioning regimens are desirable and emerging.  
 
Note the statement in #1, that for Bluebird's trials "their main toxicities, so far have been due to conditioning regimens", so obviously any process of depleting existing HSCs w/ less toxicity will be welcome, & should be helpful to incorporate into SGMO's HSC-related gene editing trials, if possible.
 
As you'll recall, these condition regimens are designed to deplete existing HSCs in bone marrow to "make room" for the engraftment of newly transplanted gene edited, HSCs.
 
 
 
 
 
 
 


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