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A New Crispr Technique Could Fix Almost All Genetic Diseases

Anzalone’s prime editor is a little different. Its enzyme is actually two that have been fused together—a molecule that acts like a scalpel combined with something called a reverse transcriptase, which converts RNA into DNA. His RNA guide is a little different too: It not only finds the DNA in need of fixing, but also carries a copy of the edit to be made. When it locates its target DNA, it makes a little nick, and the reverse transcriptase starts adding the corrected sequence of DNA letter by letter, like the strikers on a typewriter.


A less error-prone DNA editing method could correct many more harmful mutations than was previously possible.

Swiss army knife for genome research

It is the dream of every molecular geneticist: an easy-to-use program that compares datasets from different cellular conditions, identifies enhancer regions and then assigns them to their target genes. A research team led by Martin Vingron at the Max Planck Institute for Molecular Genetics in Berlin has now developed a program that does all of this.

“DNA is pretty boring, since it is practically the same in every cell,” says Martin Vingron, director and head of the Department of Bioinformatics at the Max Planck Institute for Molecular Genetics in Berlin. “While the genome is like the book of life, I am most interested in the side notes.”

These “notes” are small chemical marks attached to the DNA molecule that do not alter the genetic information itself, but influence what happens to the DNA at the respective site. In other words, these marks have an epigenetic effect. They serve as regulators of genomic regions that are responsible for the activation and deactivation of , such as promoters and enhancers.

DNA Testing: The Disconnect Between Patients and Researchers | WSJ

As genetic sequencing becomes more widespread, a disconnect is emerging between what individual patients expect to get back and what scientists are willing and able to tell them. WSJ visited MIT’s Broad Institute to learn about the murky world of genomic research data.

Photo: angela weiss/afp via getty images

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A Crispr Milestone Hints at a Future of Cures — and Oversight Concerns

Just seven years after scientists announced the first use of Crispr-Cas9 gene editing technology on human cells, researchers shared new evidence this week that Crispr can be used to cure two serious genetic disorders.

On Tuesday, NPR reported that a patient in Nashville had seen a dramatic decline in her symptoms of sickle cell disease after receiving a single gene therapy treatment in July. Sickle cell, which can lead to inflammation, debilitating pain, and life-threatening circulatory problems, affects millions of people around the world.

That same day, the biotech companies behind the sickle-cell treatment, Crispr Therapeutics and Vertex, also shared promising results from their first attempt to cure a case of beta thalassemia, another genetic disorder that affects blood proteins. Nine months after receiving the experimental treatment, a patient in Germany with beta thalassemia has almost no signs of the disorder.

Silicon Valley: The Research for Living Longer | Longevity Road Trip | TRACKS

Husbands Ian and Leon discuss the future of longevity technology and genetic research in Silicon Valley with the infamous bio-tech renegade Aubrey DeGrey.

Ian and Leon then drive a classic car down the Pacific Coast Highway and into the desert to the 7th Day Adventist community of Loma Linda to learn about “Blue Zones” and how anyone can make simple lifestyle changes that would allow them to live radically longer lives.

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TRACKS publishes unique, unexpected and untold stories from across the world every week.

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