CRISPR-based techniques allow the reconstruction of the “family tree” of the cells that compose an animal’s body by marking them with a pattern of deletions and insertions. This kind of barcoding has already helped trace embryo growth and organoid development and is shedding light on essential oncology questions by catching cancer in the act. Read how “Single-cell lineages reveal the rates, routes, and drivers of metastasis in cancer xenografts” in this Science paper and the news from Whitehead Institute.
A paper published in Nature by CRISPR innovator David Liu and a giant in medical genetics, Francis Collins, raises great hopes for treating a rare, devastating pediatric disease causing premature-aging (Hutchinson-Gilford progeria syndrome). “The outcome is incredible,” according to gene-therapy researcher Guangping Gao. “Dance on the lab bench” amazing, according to editing pioneer Fyodor Urnov. Let’s be clear: the CRISPR variant called a base-editor has helped only progeria mice so far, but results are beyond anyone’s wildest expectations. One injection is enough to fix the single-letter mutation in several tissues, doubling mice’s lifespan. To learn more, see David Liu’s tweets and the NIH Director’s Blog.
December is time for rankings and forecasts. Let’s start with STAT News celebrating young talents who could become the next generation of scientific superstars. Three CRISPR researchers appear among STAT wunderkinds. As a postdoc at the Broad Institute, Andrew Anzalone helped make a key advance by developing prime editing, where the same RNA molecule specifies the target and the desired edit. Jennifer Hamilton, from Berkeley, works on solving one of the major hurdles of CRISPR-based therapies: delivering the genome editor to the desired cells. Cameron Myhrvold, has since worked at the Broad Institute on developing CRISPR-based diagnostics such as CARMEN and is about to start his own lab at Princeton.
The Nobel Prize for CRISPR is one of the most exciting ever assigned in chemistry and one of the most celebrated in the media, for reasons related to the invention and the inventors alike. On the one hand, the technique is changing the practice and the image of genetic engineering. On the other hand, Jennifer Doudna and Emmanuelle Charpentier are not merely great scientists; they are a success story in cracking the glass ceiling and a symbol of the strength of collaboration.Continue reading
Covid19 is affecting everyone, but it has hit the sickle cell (SCD) community particularly hard. According to STAT News the pandemic has temporarily stopped clinical trials and the introduction of new drugs, besides directly impacting SCD patients who are at high risk for severe complications from Sars-Cov2 infection and may need hospital assistance for SCD pain crises.Continue reading
Consider this scenario, depicted in Nature a few years ago. “It’s 2037, and a middle-aged person can walk into a health centre to get a vaccination against cardiovascular disease. The injection targets cells in the liver, tweaking a gene that is involved in regulating cholesterol in the blood. The simple procedure trims cholesterol levels and dramatically reduces the person’s risk of a heart attack”.Continue reading
Click the links below to discover Ddda, the exceptional enzyme that allows mitochondrial editing, and celebrate curiosity-driven research.
The Nature paper by Joseph Mougous and David Liu: “A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing”
The news: “Scientists make precise gene edits to mitochondrial DNA for first time”
The news and views: “Mitochondrial genome editing gets precise”
The editorial: “Mitochondrial genome editing: another win for curiosity-driven research”
COVID-19 overshadowed CRISPR’s advancements this year. The July issue of Nature Biotechnology keeps you up with the latest news and trends in genome editing, covering clinical testing, tools, patents, and more.
CRISPeR Frenzy asked Luigi Naldini of the San Raffaele Telethon Institute for Gene Therapy in Milan for comment on three studies published in June on the preprint server bioRxiv. The experiments were carried out independently by the groups of Kathy Niakan of the Francis Crick Institute in London, Dieter Egli of Columbia University in New York City, and Shoukhrat Mitalipov of Oregon Health & Science University in Portland. These findings heighten safety concerns about heritable genome editing (see the news item by Heidi Ledford in Nature). Below you can read Naldini’s thoughts.Continue reading
Victoria Gray is the first US patient treated with CRISPR for a genetic disorder. Don’t miss the interview she gave to NPR almost one year after the experimental treatment.