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The Dawn of a New Era: First Approval of Genome Editing and Its Future Potential

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Mason Walker
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The Dawn of a New Era: First Approval of Genome Editing and Its Future Potential

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Marking a historic medical milestone, the U.S. Food and Drug Administration (FDA) has given its first ever approval for a CRISPR gene-editing treatment for sickle cell disease. This approval, although indirect, disruptive, and rudimentary, is a testament that genome editing is just getting started, with the potential to revolutionize the field of medicine.

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First CRISPR Gene-Editing Treatment Approval

The approved treatment, known as exa cel, involves editing a gene crucial to the shape and function of red blood cells, and appears to provide a functional cure for sickle cell disease for at least one year. The treatment has been tested in around 100 people with either sickle cell anemia or beta thalassemia and has shown no major adverse health impacts. However, it can cause side effects such as nausea and fever. Apart from exa cel, another gene therapy named lovo cel has also secured FDA approval, and numerous studies are exploring other types of gene therapies for sickle cell anemia and beta thalassemia.

CRISPR Cas9 Therapy – A Breakthrough in Genome Editing

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The approval of the CRISPR Cas9 therapy for sickle cell disease is not just a breakthrough in itself but also opens the door for the next generation of genome editing techniques that offer more precision and versatility. These innovative systems include base editing, prime editing, and epigenome editing.

Base Editing, Prime Editing, and Epigenome Editing

Base editing, which allows for the alteration of individual DNA letters, is currently being used in clinical trials for high cholesterol and leukemia treatment. Prime editing, known for its flexibility, can target and correct virtually any site in the genome. It is being made more efficient and is under development for clinical trials to treat genetic immune disorders. Epigenome editing, on the other hand, can modify how genes are expressed by changing the epigenome. However, its progress has been slower than other editing techniques due to assumptions about edits being erased during cell division.

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The Future of Genome Editing

In the wake of this landmark decision, a new type of medicine that can tackle genetic conditions that are hard to treat is being heralded. The approval of these gene therapies, including the first ever CRISPR gene editing treatment, signals the beginning of a new era for gene editing medicine. The treatments involve an intensive process aimed at fixing the offending genes that cause the disease at the genetic level. Both treatments have demonstrated impressive clinical trial results with few safety concerns. However, long-term monitoring is necessary to measure their lasting power and safety.

This milestone approval of the first CRISPR treatment in the U.S. for sickle cell disease showcases the immense potential of genome editing. It involves editing mutated genes in a one-time procedure that boosts the production of fetal hemoglobin, which can assist healthy blood cells in outcompeting the sickled versions. The most dangerous side effect of CRISPR is off-target editing, while the biggest concern for the other therapy is where the gene for the healthy hemoglobin is inserted. Both companies have been asked by the FDA to follow patients for 15 years to document any potential long-term effects of the therapies.

In the words of David R. Liu, the recent approval of genome editing signals a new dawn in medical science. With more research and clinical trials, we can expect to see more advancements in this field, transforming the way we approach and treat a wide array of genetic conditions.

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