The Effective Treatment for Lymphoblastic Leukemia

One-year-old Layla was diagnosed with acute lymphoblastic leukemia when she was three months old, and after endless amounts of chemotherapy and a failed bone marrow transplant, her parents were informed that nothing else could be done. Not willing to give up their baby daughter so easily, Layla’s family agreed to put Layla through a new clinical trial involving a form of gene therapy, referred to as gene editing, in 2015. Within a month of receiving treatment, the genetically engineered cells killed off the cancerous cells in Layla’s bone marrow, making Layla the first person to be saved by gene editing.

The scientific breakthrough of genome editing has made an incredible impact on the options of treatment available for people with diseases that previously were claimed unable to be cured. Genome editing opens up the ability to treat people predisposed to expressing a genetically inherited illness. This treatment is targeted to be able to treat diseases like cystic fibrosis, sickle-cell anemia, Duchenne Muscular Dystrophy, Alzheimer’s disease, and more.

In addition to genetic illnesses, gene editing can be used on embryos to stop and prevent detrimental genetic diseases that are discovered before birth. Genome editing should become an accepted form of treatment because of the opportunities the treatment gives to people who could not receive any form of cure before.

The process of genome editing is commonly associated with the CRISPR-Cas9 system. As described by the National Institute of Health, the system works by creating a piece of RNA with a guide sequence that attaches to a specifically targeted sequence of DNA.

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When the RNA binds to a Cas9 enzyme, it creates “CRISPR” and is able to split the DNA at the targeted location. After the DNA is cut, the mutation present can be adjusted by adding, deleting, or replacing a part of the DNA sequence. With the ability to correct unwanted mutations in the DNA, the ability to treat people suffering from inherited, incurable diseases becomes possible.

In addition to this, the CRISPR method of treatment is one that is easily accessible and fairly cheap. As stated by Jennifer Doudna, a biochemist who is known for her extensive work done involving CRISPR, “…the opportunity to take a technology like CRISPR and utilize it has some appeal, because of its relative simplicity.” (Doudna, 2015) Doudna relates CRISPR technology to a “software for the genome,” because, “we can program it easily…with RNA”.

There are many positive advancements that correlate to the use of the CRISPR system, but some people are wary of the ethical implications behind it. The main issue that makes CRISPR so controversial is how easily it could get out of hand. When regulated too loosely, CRISPR can be used to edit an embryos’ genes to meet specific preferences and change natural traits, such as eye color, height, and athletic ability. Under certain circumstances, CRISPR can be incredibly life changing and beneficial, which is why it would be important to have specific criteria one would have to meet to qualify for it.

Soon after Layla, another baby girl was given the CRISPR treatment for leukemia when she was 16 months old. As of now, both girls are healthy and cancer-free because of this transformative treatment. The use of CRISPR and genome editing should be widespread because of the immense benefits it would create for those who didn’t have options before.