First sickle cell patient to live better in 2020 after CRISPR treatment

Sickle cell anemia is an inherited disorder caused due to a mutation in the hemoglobin, and it creates havoc on patients. People with this disorder have atypical hemoglobin molecules called hemoglobin S. It transforms the red blood cells into a sickle cell shape limiting their ability to carry oxygen and ultimately results in anemia and stroke. The treatment that reverses this condition is the bone marrow transfusion that requires a donor, but it works only for just 10% of the patients.

Victoria Gray, a Mississippi woman, suffered from sickle cell anemia from her young age. She volunteered herself for one of the most anticipated medical experiments in decades. She was the first person in the U.S. to receive the CRISPR treatment, a gene-editing technique one year ago that could change a person’s cell.

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What is CRISPR technique

CRISPR, which stands for Cluster Regularly Interspaced Short Palindromic Repeats, is a type of gene editing that makes the scientists cut and paste genes into DNA. It is based on a targeted DNA-destroying defense system originally found in prokaryotes. In the lab, researchers can create a small piece of RNA which effectively binds to the targeted DNA in a genome. Cas9 protein is highly utilized in genetic engineering as it helps in cleaving the DNA in the cell. Cas9 is found in certain bacteria that are capable of attacking the viral DNA for its defense. These proteins are bound to the small piece of RNA created in the lab. As a whole, this sequence recognizes the DNA pattern, and the Cas9 enzyme cuts the DNA in the mutated location. Researchers use the cell’s DNA repair machinery to add or delete pieces of genetic material or to make changes to the DNA by replacing the existing segment with a customized DNA sequence.

Doctors removed bone marrow cells from Gray’s body and edited a gene inside them with CRISPR to produce normal hemoglobin and introduced the modified cell back into her body last July. Scientists called the technique as the genetic equivalent of Microsoft Word.

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It is found that Gray responds well to the treatment, and she did not suffer from any unnecessary pain after the editing. Even she did not receive any blood transfusion for one year. The doctor said that the one year period is too early to decide whether the CRISPR treatment is efficient and definite to sickle cell disease. Altogether, there was good improvement in the health of the women, and she even was chronicled by NPR.

The woman is thankful for the treatment given by the doctors, and she now cares for her three children during this COVID-19 pandemic and also the racial injustice protests in the United States. Without such treatment, it would be highly impossible to be with her family as she might have been hospitalized amid the pandemic.

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Source: HuffPost India

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