How Is Sickle Cell Research Using Gene Editing?
More Programs Featuring Maya Bloomberg
In this program:
Sickle cell patients may benefit from some medical advances like gene editing and gene therapy. Hematology nurse practitioner Maya Bloomberg defines gene editing versus gene therapy and explains gene editing research that is examining treatment advances for sickle cell patients.
Transcript
Interviewer:
So what is the difference with the gene editing and the gene therapy?
Maya Bloomberg, APRN:
That’s a very good question. So gene editing is when you're actually editing a person's gene, where gene therapy is we're putting an external vector, so for sickle cell studies, it's actually a gene editing. We’re not doing gene therapy, and there are different concepts behind gene editing, you can have a gene editing where we're going to turn off a switch. So fetal hemoglobin correction, you mentioned your son has very high fetal hemoglobin, so babies are born with fetal hemoglobin at around six months of age, that's when we stopped fetal hemoglobin production, and that's when we start producing our adult hemoglobin. So hemoglobin A, if you don't have sickle cell hemoglobin B with sickle cell, so that's why we start seeing issues in sickle cell patients that are on 6 to 12 months of age, because we lose that fetal hemoglobin production, there are some people who have an actual mutation where their body just never turns that off switch on. So they continue to produce fetal hemoglobin that hereditary persistent fetal hemoglobin. But now we're looking at gene editing to do the same thing is essentially keep that switch on so your body never switches it off, and you continue to produce fetal hemoglobin. So that's one area where you can do gene editing, another is using gene editing to correct the actual mutation itself with sickle cell instead of...
You have that one gene instead of indocin, tyrosine; and therefore, you produce sickled hemoglobin so instead of hemoglobin, now we're looking at using CRISPR to try to introduce hemoglobin A, so your body over time will continue to produce hemoglobin and that I can go...and that's the going to the Bluebird’s trial, which was a hold for a little bit. But now it's back on since they had some adverse events, but that's the point of research we want to find out, is it safe, is it effective before we have a commercially available...so it's definitely far off into the future for when that's going to be available. But the interim data that we have thus far, it's really exciting, and I think it's going to be a game-changer for sickle cell patients.
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