A possible cure for sickle cell?



Red blood cells with sickle/crescent shaped red blood cell anemia are inherited blood disorders. It results in a lot of infections, swollen hands and legs, pain, extreme exhaustion, and delayed puberty or growth. Treatment usually focuses on symptom management and may involve blood transfusions, antibiotics and immunizations to avoid bacterial infections, painkillers during acute crises, and hydroxyurea to lessen the frequency of pain attacks.

The New England Journal of Medicine published a new study (Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Condition/Kanter et al.) that, if applicable, could lead to a cure for this terrible disease.

Martin Steinberg, MD, a professor of medicine at Boston University School of Medicine, offers commentary on the study's findings in an editorial published in this week's issue of the New England Journal of Medicine. This was the first successful gene therapy study for sickle cell disease, adding a gene that prevents complications to patient blood stem cells. According to Steinberg, a hematologist and geneticist at Boston Medical Center, "gene therapy with autologous stem cells expands the potential of a cure to all patients without the requirement for immunosuppression."

Steinberg notes that even though the study participants' sickle cell symptoms had disappeared, the majority of them still had red blood cells that had shorter than average lives, which he believes may be linked to certain issues in the long run.

According to Steinberg, the treatment must be curative or almost curative and last a lifetime in order for patients to embrace it. "At this time, we are unsure of the results' long-term viability, but based on this study, the prospects appear promising," he claims.

Steinberg agrees that the majority of sickle cell disease patients worldwide would not experience improved health as a result of any highly effective gene treatment. "The majority of those who suffer from this disease reside in Africa and India, where access to highly advanced medical care is scarce. More medications that can be given orally and raise fetal hemoglobin levels are required. The communities most affected by this disease will more likely benefit from this."

Boston University School of Medicine

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