Asked by: Atiq Hiraldo
asked in category: General Last Updated: 14th January, 2020

Can Crispr cure sickle cell?

The first CRISPR gene therapy to cure sickle-cell disease. In 2019, CRISPR gene-editing therapy was used for the first time to treat sickle cell disease. Hematopoietic stem cell (HSC) transplantation is the only curative, nonexperimental option for patients with severe forms of the disease.

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Keeping this in consideration, how can Crispr be used to cure sickle cell disease?

There are two main ways that CRISPR-Cas9 is being used to treat sickle cell disease. One approach consists of repairing the gene for hemoglobin S in order to cause the normal form to be produced. The second approach involves replacing hemoglobin S with hemoglobin F.

Similarly, can Crispr cure muscular dystrophy? A study has shown that CRISPR can be used as a regenerative technique to treat Duchenne muscular dystrophy, which could be developed as a therapeutic option for humans. Researchers have successfully demonstrated in a mouse model that CRISPR can regenerate muscle suffering from Duchenne muscular dystrophy (DMD).

Correspondingly, could gene therapy cure sickle cell anemia?

When the cell repairs its DNA, the gene no longer works and more fetal hemoglobin is produced. Gene therapy offers an advantage over bone marrow transplant, in that complications associated with a bone marrow donation — now the only cure for the disease — such as finding the right match are not a concern.

Does fetal hemoglobin help sickle cell?

New HHMI research shows that reactivating fetal hemoglobin production in adult mice effectively reverses sickle cell disease. Not long after birth, human babies transition from producing blood containing oxygen-rich fetal hemoglobin to blood bearing the adult hemoglobin protein.

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