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Comment
. 2021 Feb 3;29(2):416-417.
doi: 10.1016/j.ymthe.2021.01.009. Epub 2021 Jan 19.

A "Shot in the Arm" for Sickle Cell Disease

Tim M Townes  1
Affiliations
Comment

A "Shot in the Arm" for Sickle Cell Disease

Tim M Townes. Mol Ther. .
No abstract available

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Figures

Figure 1
Figure 1
Gene Addition and Gene Editing to Correct Sickle Cell Disease In vivo hematopoietic stem cell transduction with HDAd5/35++ vectors containing modules for SB100x-mediated γ-globin gene addition (left square and circle) and CRISPR-Cas9-triggered reactivation of γ-globin (right square and circle) conferred γ-globin levels that were curative in a humanized mouse model of sickle cell disease.
See this image and copyright information in PMC

Comment on

References

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