Using CRISPR gene editing, researcher Arkasubhra Ghosh set out to save young Uditi Saraf immediately. He wanted to use a new method to stop her brain cells from making harmful proteins.
(Photo: Unsplash/ Sangharsh Lohakare )
Racing Against Time
Even though they faced big problems, Ghosh and his team moved quickly forward. Finally, in June, they told Uditi's parents that the therapy might not happen for another six months. But Uditi's mother told them to work faster because she knew how bad her daughter's situation was.
When Ghosh saw Uditi at the breakfast table, everything became clear. It was almost impossible for the girl to see or hear, and she couldn't walk or feed herself.
Ghosh felt so bad that he returned to his lab at Narayana Nethralaya Eye Hospital in Bengaluru, India, vowing to do everything he could to save her. Uditi died four months later, which was very tragic.
Uditi's family has been facing significant challenges due to her rare disease, familial encephalopathy, with neuroserpin inclusion bodies (FENIB). They experimented with various approaches, including ayurvedic medicine and specialized diets, and even relocated to upstate New York for improved academic support. Despite their efforts, Uditi's health continued to deteriorate.
Orrin Devinsky, an epilepsy specialist in New York, recommended that CRISPR genome editing be helpful. This approach gave people hope even if it had not been tested clinically. The Sarafs funded early research, but due to the COVID-19 pandemic and Uditi's deteriorating health, they had to return to India.
They traveled to India and talked with Debojyoti Chakraborty, a geneticist who has researched using CRISPR for sickle-cell disease treatments. The team persevered despite encountering challenges such as obtaining governmental approvals and facing issues during the experiments. By June 2023, they were on the verge of conducting experimental trials on mice for their treatment.
October brought Uditi pneumonia, which claimed her life shortly after. Despite the terrible defeat, Ghosh and his group are still committed to their objective. They were in agony, recalls Chakraborty.
The Promise of CRISPR Genome Editing
With CRISPR gene editing, researchers may alter an organism's DNA. This technique allows genetic material to be added to, removed from, or altered at particular places in the genome. One well-known tool in this sector, CRISPR-Cas9, is praised for being faster, more accurate, and less expensive than earlier gene editing methods.
The CRISPR-Cas9 technique was the initial approach utilized to protect microorganisms from illnesses. The RNA fragments eliminate the threat by cleaving the virus DNA. Scientists have used this technology to modify the DNA of several organisms, including humans. Researchers can achieve the necessary genetic alterations by utilizing the Cas9 enzyme to cleave the DNA following the creation of a guide RNA specifically targeting particular areas of the DNA.
Treating hereditary disorders holds great promise for CRISPR-Cas9. Though yet a research concept for many applications, it offers hope to those with sickle cell disease, hemophilia, and cystic fibrosis. However, its application also begs ethical concerns, particularly about DNA editing, which alters future generations and is now illegal in many countries.
India is known for making expensive, complicated drugs, and Ghosh thinks this will also be true for gene treatments. His lab wants to get treatments to people in other countries for much less than they cost in the U.S. Ghosh says, "We will definitely short-circuit this whole field."
At Uditi's funeral, Rajeev thought about how impatient she was. "She could not let science win," he said. "She was always ahead."
These advancements exemplify the possibility of personalized pharmaceuticals, such as CRISPR-based therapeutics for uncommon disorders. Scientists persist in developing platforms for producing these therapies as they believe each progress can potentially enhance the quality of life for those with rare genetic illnesses.
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