By Joe Solovey
November 2024
Within just the past few months, the world of CRISPR has seen some revolutionary breakthroughs. Since 2012, when CRISPR became a widespread gene editing tool, there have been numerous uses for the treatment. Within research, these uses extend to generating cells with specific mutations to find potential causes of diseases such as cancer.
Revolutionizing Cancer Research with CRISPR
In Italy, a paper published on November 14 utilized this very mechanism. In their research, Luca Gelsomino and his group of researchers at the University of Calabria induced a mutation into breast cancer cells using CRISPR. This mutation, known as the Y537S-Era mutation, was found to affect the tumor microenvironment. These effects on the tumor microenvironment helped to induce healthy fibroblasts into cancer-associated fibroblasts (CAFs). The findings of this paper help show a possible method that breast cancer cells use to induce metastasis in breast cancer. Through CRISPR, this mutation can be easily induced into cells, making discoveries such as this possible.
Advances in CRISPR Gene Delivery: Sendai Virus
A new potential modification of CRISPR was published in the American Society of Microbiology on November 4, 2024, from the Icahn School of Medicine at Mount Sinai. This innovation used an RNA virus called Sendai virus (ts SeV) as the vector for delivering the CRISPR-Cas9 gene editing tool. This mechanism provided a success rate of over 90% in the HIV-1 cells studied. This new vector has also been shown to induce less of an immune response than the current DNA virus vector used in CRISPR. These modifications can be incorporated into future therapies reducing fewer side effects in patients.
Gene Therapy Breakthroughs for Duchenne Muscular Dystrophy
In the world of CRISPR-based therapies, CRISPR has potential far beyond the recently FDA-approved sickle cell disease gene-editing treatment, Casgevy. Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the dystrophin gene, essential for muscle stability and repair. Affecting mainly males, this condition leads to progressive muscle degeneration, with many individuals losing the ability to walk by adolescence. Without intervention, life expectancy is often limited to early adulthood, primarily due to respiratory or cardiac failure. Recent advancements in CRISPR technology have introduced hope for those affected by DMD. Using precise gene-editing tools, scientists aim to correct mutations in the dystrophin gene. In research published in September, preliminary trials performed by the University of Alberta have shown promising results, with significant improvements in muscle strength and overall health when paired with oral supplements. These treatments not only extend life expectancy but also improve quality of life, offering a potential cure for this debilitating disease. Ongoing research continues to optimize these therapies, moving closer to clinical implementation
Recent research in the field of CRISPR has shown much advancement along diverse avenues. From unlocking mechanisms of breast cancer metastasis to innovations in CRISPR technology itself, as well as new hope for curing debilitating diseases, the field of CRISPR is rapidly growing for the better.
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