Tag Archives: Gene-editing

CRISPR-SKIP, New Gene Editing Technique

What if doctors could treat previously incurable genetic diseases caused by errors or mutations in genes? Thanks to new research by American scientists at the University of Illinois, we are one step closer to making that a reality. Published in Genome Biology, their work is based on CRISPR-Cas9, a groundbreaking genome editing system.

Typically, cells in the body “readDNA to produce the proteins needed for different biological functions. . Scientists can change how the DNA is read using CRISPR gene-editing technology. CRISPR-Cas9 is often used to cut out specific areas of DNA and repair faulty genes. In the current study, the researchers modified existing technology to create CRISPR-SKIP. Instead of breaking DNA to cut faulty genes out, CRISPR-SKIP changes a single base of the targeted DNA sequence, causing the cell to skip reading that section of DNA.

According to the study authors, CRISPR-SKIP can eliminate faulty sections of DNA permanently, allowing for long-lasting treatment of some genetic diseases with one treatment. They successfully tested their technique in cell lines from both mice and humans. The scientists aim to test the method in live organisms in the future.

CRISPR-SKIP has the potential to help treat many diseases such as cancer, rheumatoid arthritis, Huntington’s disease, and Duchenne muscular dystrophy to name a few. Because the method only requires editing of a single base, it is simple, precise, and adaptable to a variety of cell types and applications.

Source: https://news.illinois.edu/
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https://www.medicalnewsbulletin.com/

CRISPR Reverses Duchenne Muscular Dystrophy Mutation

CRISPR-Cas9 has, for the first time, been tested by systemic delivery in a large animal—and the results are striking. Working in a dog model of Duchenne muscular dystrophy (DMD), the gene editing not only restored the expression of the protein dystrophin, it also improved muscle histology in the dogs.

Our technology was developed using human cells and mice to correct the same type of mutation as in these dogs. It was critical for us to test gene editing in a large animal because it harbors a mutation analogous to the most common mutation in DMD patients,” said Eric Olson, Ph.D., professor and chair of molecular biology at the University of Texas Southwestern Medical Center and lead author. The researchers wrote that this is “an essential step toward clinical translation of gene editing as a therapeutic strategy for DMD.”

Indeed, Dame Kay E. Davies, Ph.D., professor of anatomy and director of the MRC Functional Genomics Unit at the University of Oxford and a pioneer in the field of DMD research, echoes this sentiment explains, “This is a very exciting paper as it shows that gene editing can be reasonably affective in a large animal model of DMD.”

The paper, “Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy,” appears in the last issue of Science.

Source: https://www.genengnews.com/

How To End Malaria

Gene-editing technologies that alter mosquitoesDNA could prove critical in the fight against malaria, Bill Gates said on Wednesday, and ethical concerns should not block progress in such gene-modifying research.

Speaking at the Malaria Forum conference in London, the billionaire Microsoft co-founder and philanthropist said that while gene editing raises “legitimate questions”, that should not jeopardize exploration of tools such as CRISPR gene editing and so-called “gene drive” technologies.

I’m very energized about the potential of gene drive. (It’s) the kind of breakthrough we need to support,” Gates said. “It may prove critical here.” 

Gene drive technologies alter DNA and drive self-sustaining genetic changes through multiple generations by overriding normal biological processes. CRISPR technology enables scientists to find and modify or replace virtually any gene. The techniques are being explored across science – from human medicine to livestock– and crop-breeding. In mosquitoes that transmit malaria, genetic alterations can be used to induce infertility to reduce populations, or alter the insects’ ability to carry and pass on the malaria parasite. 

The technologies can be extremely powerful, but they are also controversial, since such genetically engineered organisms released into the environment could have an unknown and irreversible impact on the ecosystem. Asked in a interview with Reuters about that controversy, Gates said there were understandable concerns about safety and efficacy that would need to be addressed in research and trials. But he countered: “Malaria itself is quite controversial – it kills about 400,000 kids a year. So we’re definitely not on the side of malaria.”

He also noted that at their summit in January, leaders of the African Union endorsed gene drive research as part of the fight against a disease that continues to kill their people.

Source: https://www.reuters.com/