Research and publish the best content.
Get Started for FREE
Sign up with Facebook Sign up with X
I don't have a Facebook or a X account
Already have an account: Login
Genetic Engineering Publications - GEG Tech top picks
36.6K views |
+0 today
 Your new post is loading...
Your new post is loading...
|
Scoop.it!
An international, peer-reviewed genome sciences journal featuring outstanding original research that offers novel insights into the biology of all organisms
BigField GEG Tech's insight:
The authors combined CRISPR/Cas9 technology with the piggyBac transposon to efficiently correct the HBB mutations in patient-derived iPSCs without leaving any residual footprint. No off-target effects were detected in the corrected iPSCs, and the cells retain full pluripotency and exhibit normal karyotypes. When differentiated into erythroblasts gene-corrected iPSCs restored expression of HBB compared to the parental iPSCs line.
|
Here, the central role of BCL11A in control of HbF is reviewed from the perspective of how findings may be translated to gene therapy in the not-too-distant future. This summary traces the evolution of recent studies from the initial recognition of BCL11A through GWAS to identification of critical sequences in an enhancer required for its erythroid-specific expression, thereby highlighting an Achilles heel for genome editing.