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Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
In sickle cell disease, a gene mutation causes the hemoglobin molecules ... It does so by suppressing the gene BCL11A, which normally shuts down fetal hemoglobin production after birth.
The Scientist5y
Early Results Are Positive for Experimental CRISPR Therapies
The therapy, known as CTX001, is applied to blood stem cells removed from the patient to cleave the BCL11A gene that represses the ... to overcome problems associated with mutations in the gene ...