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Ther Adv Hematol 29(11):2040620720955000 Sagi V, Mittal A, Tran H, Gupta K (2021) Pain in sickle cell disease: current and potential translational therapies

Since there are deficiency alleles with mutations outside the coding sequence and there are individuals with three mutations, it is not possible to assume that all coding region mutations are responsible for the disease

However, several reports highlight that while generally safe, these injections can sometimes cause adverse effects, most notably hypersensitivity and anaphylactic reactions

[194] Importantly, the specific and often heterogeneous contexts surrounding genetic targets during the screening process may complicate clinical translation of preliminary findings