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These results emphasize the complex regulatory pathways involved in HbF generation and demonstrate the potential of CRISPR-Cas9 as a therapeutic tool for treating haemoglobin disorders. The discovery opens up new possibilities for gene therapy by showing that even small deletions in the BCL11A enhancer can effectively revert the switch from adult to foetal haemoglobin.
These results highlight the complex regulatory pathways involved in HbF generation and demonstrate the potential of CRISPR-Cas9 as a therapeutic tool for the treatment of haemoglobin disorders. This discovery opens new possibilities for gene therapy by showing that even small deletions of the BCL11A enhancer can effectively reverse the transition from adult haemoglobin to foetal haemoglobin.
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