Harnessing developmental biology to achieve safe and efficient in vivo genome editing of HSCs
Grant Award Details
Grant Type:
Grant Number:
DISC0-17394
Investigator(s):
Award Value:
$2,316,683
Status:
Pre-Active
Grant Application Details
Application Title:
Harnessing developmental biology to achieve safe and efficient in vivo genome editing of HSCs
Public Abstract:
Research Objective
We seek to develop a more accessible method of genome editing of blood stem cells directly in the body, so that the treatment can be safer and less expensive than the currently available approaches.
Impact
We think our new protocol could transform the care of children with blood diseases like Sickle Cell Disease, thalassemias, and metabolic conditions.
We seek to develop a more accessible method of genome editing of blood stem cells directly in the body, so that the treatment can be safer and less expensive than the currently available approaches.
Impact
We think our new protocol could transform the care of children with blood diseases like Sickle Cell Disease, thalassemias, and metabolic conditions.
