He Jiankui’s original research, published for the first time, could have failed, scientists say.
Category: genetics – Page 361
Molecular biologist Fiona Behan reports on how the CRISPR gene-editing technique can find cancer’s weak points, identifying targets for new drugs.
Editing of hemoglobin gene leads two people with different conditions to no longer need regular blood transfusions.
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.
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 genes, such as promoters and enhancers.
Biochemical makeover allows Escherichia coli to use carbon dioxide as a building block for its cells.
Following strong results for its flagship CTX001 drug, should you add CRISPR to your portfolio?
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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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.