#sicklecellawarenessmonth Day 18
Let’s talk about #GeneTherapy and curing #sicklecell
Are we there yet?
Well, almost
We are way closer than we have ever been and there’s hope that we can truly offer a cure for SCD soon
A short 🧵 with more details…
1/5
Let’s talk about #GeneTherapy and curing #sicklecell
Are we there yet?
Well, almost
We are way closer than we have ever been and there’s hope that we can truly offer a cure for SCD soon
A short 🧵 with more details…
1/5
#GeneTherapy refers to various methods to edit or change genes to either fix a mutation or induce other beneficial genetic changes to treat a condition
For #BoneMarrowTransplant, you need a donor (and <20% of Warriors have donors) but for #GeneTherapy you are your own donor
2/5
For #BoneMarrowTransplant, you need a donor (and <20% of Warriors have donors) but for #GeneTherapy you are your own donor
2/5
Before having edited genes infused back, m chemotherapy medications (usually Busulfan) are required to kill current bone marrow and hematologic cells
There are risks including infertility, secondary cancer risk, others
Risk must always be balanced against potential reward
3/5
There are risks including infertility, secondary cancer risk, others
Risk must always be balanced against potential reward
3/5
Now there are 2 leaders
@bluebirdbio has a product already approved for #thalassemia using virus to ⬆️ anti-sickling hemoglobin - there are a few cases of leukemia after #GeneTherapy that aren’t 100% well understood
@VertexPharma uses #CRISPR to edit genes and ⬆️ HbF
4/5
@bluebirdbio has a product already approved for #thalassemia using virus to ⬆️ anti-sickling hemoglobin - there are a few cases of leukemia after #GeneTherapy that aren’t 100% well understood
@VertexPharma uses #CRISPR to edit genes and ⬆️ HbF
4/5
These methods are expensive and complicated and won’t be available in places like #Africa but @gatesfoundation has invested in finding a way to do this more simply with “one shot”
Even in the US, we need to work hard to ensure that cures are truly accessible when approved
5/5
Even in the US, we need to work hard to ensure that cures are truly accessible when approved
5/5
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