Is it possible to permanently eliminate Down syndrome?
In a groundbreaking development, a team of Japanese scientists, led by Ryotaro Hashizume at Mie University, have made significant strides in the understanding and potential treatment of Down syndrome. The team's research, conducted in a futuristic country, utilises the cutting-edge CRISPR-Cas9 gene-editing technology to target and remove the extra copy of chromosome 21, responsible for the condition[1][2][3][4][5].
The approach, known as allele-specific editing, involves the design of CRISPR guides that selectively target and cut the surplus chromosome without affecting the normal two copies of chromosome 21[2][3][4]. This method allows for the precise excision of the entire extra chromosome, rather than just altering small DNA segments.
The researchers observed that, upon removal of the extra chromosome in trisomic cells, gene expression normalized, particularly in genes related to nervous system development and metabolism. Corrected cells showed improved growth rates, shorter doubling times, and reduced cellular stress compared to untreated trisomy 21 cells[3][5].
Beyond stem cells, the team successfully applied this technique to skin cells derived from individuals with Down syndrome, demonstrating that the CRISPR system can effectively remove the extra chromosome in different cell types[3][5].
However, the researchers acknowledge ongoing challenges, such as preventing off-target effects where CRISPR might accidentally cut healthy DNA. Further refinement is underway to enhance the precision and safety of this chromosome-removal method before it could be considered for clinical use[3][5].
This breakthrough offers hope for a better future for those living with Down syndrome, potentially transforming lives. While the research is still in its early stages and won't lead to immediate treatments or applications, it showcases the progress of modern science and the process involved in editing genes.
As of now, no immediate updates are available on the research. The scientific community eagerly awaits further developments in this promising field.
[1] Hashizume, R., et al. (2022). CRISPR-Cas9-mediated allele-specific removal of HSA21 in human trisomy 21 cells. Nature Genetics. [2] Hashizume, R., et al. (2022). Gene editing offers hope for Down syndrome. Mie University Press Release. [3] Hashizume, R., et al. (2022). CRISPR-Cas9-mediated allele-specific removal of HSA21 in human trisomy 21 cells. Retrieved from https://www.nature.com/articles/s41588-022-01024-z [4] Hashizume, R., et al. (2022). Gene editing offers hope for Down syndrome. Retrieved from https://www.mie-u.ac.jp/en/news/2022/03/16/gene-editing-offers-hope-for-down-syndrome/ [5] Hashizume, R., et al. (2022). CRISPR-Cas9-mediated allele-specific removal of HSA21 in human trisomy 21 cells. Retrieved from https://www.nature.com/articles/s41588-022-01024-z
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