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Author Archives: Anna Meldolesi
CRISPR trials: the 2024 update
The recent approval of Casgevy represents the first official success of gene editing-based therapies. The treatment for sickle cell anemia and thalassemia came in record time, only 11 years after CRISPR was invented. “Two diseases down, 5,000 to go,” commented Fyodor Urnov, Director of Technology & Translation at the Innovative Genomics Institute. Among the many diseases awaiting a cure, what will be the next to benefit from CRISPR? At what rate can we expect new treatments to arrive? The periodic update published by IGI is a must-read to navigate through hope and hype, papers and press-releases. The picture is overwhelmingly positive, but there is also some cause for disappointment. Here is an excerpt from the introduction:
Continue readingCRISPR plants for all tastes
It could take a while to bring the first CRISPR products to our tables, but it is always a good time to see what progress is being made in the labs. Here are some novelties, reported by Isaaa: high-fiber barley, aromatic soy milk, extralong-grain wheat, TiGER strawberries, and my favorites: seedless, thornless, and higher-yielding blackberries and black raspberries.
Live Dance Performance – The Choreography of CRISPR
If you are in Cambridge, Massachusetts, don’t miss the live dance performance to be held at the MIT Museum on March 16th. The Choreography of CRISPR is all about “twisting, cutting, inserting, copying, repeating, palindromes, and cluster”, “an intricate dance of spiraling and folding patterns” (you can watch a minute from the première on the facebook page of the NYC-based contemporary dance company Pigeonwing Dance; choreography by Gabrielle Lamb, original music by James Budinich).
CRISPR’s next target is the fetus genome
The goal is to treat unborn children as early as possible, before their disease causes irreversible damage. But the ambition is to do so without heritable DNA changes, that is, by targeting only somatic tissues and avoiding sex cells. Fetal genome editing, then, differs from embryo editing, which has raised so much controversy in recent years. The best way to understand how far it has come and how much remains to be done is to tell the story of the scientist most committed to this challenge. The opportunity is provided by a longread published in STAT, where Tippi MacKenzie’s biography is interwoven with a review of the field.
Continue readingCRISPR plants – what the EU Parliament got right and wrong
There is no doubt that this is good news: on February 7, the European Parliament approved the Commission’s regulatory proposal on New Genomic Techniques, covering also CRISPR plants. Some of the approved amendments (particularly the one on genetic modification of polyploid plants) have the effect of improving the text, others risk being a problem and should be reconsidered during the trilogue with member states (particularly the requirement to label all final products, even if they do not contain foreign genes). The European Parliament also brought in the issue of non-patentability of NGT plants, which would deserve to be addressed elsewhere. For more information, this is the position expressed on February 14 by European Plant Science Organisation (EPSO).
A multiple sclerosis trial and more CRISPR news
Anyone interested in advanced therapies is familiar with the acronym CAR-T. These are T lymphocytes modified (also with the help of CRISPR) to better recognize and attack cancer cells, and they have already proven to be a successful strategy for blood tumors. Now hopes are high that a similar approach may also prove useful for multiple sclerosis, which is an autoimmune disease. The idea is to use CAR-Ts to prevent B lymphocytes from attacking nerve cells, including in the brain. The first clinical trial is recruiting patients in the U.S. Read more in Nature.
Let’s come to the use of New Genomic Techniques in crops. The European Commission’s regulatory proposal (approved by the EU Parliament on Feb. 7) excludes the use of edited plants in organic farming, but among organic producers not everyone is against NGTs and this may bode well for a possible peaceful coexistence between the different types of production in the years to come.
Finally, we point out the latest advance in animal editing: porcine virus-resistant pigs. The paper came out in the CRISPR Journal, but you can also read about it in GEN.
Patient-pioneer in the pantheon of medicine
According to Fyodor Urnov, she should be added to “the pantheon of names inscribed in golden letters in the history of biomedicine.” That list includes other pioneering patients such as James Phipps (the boy vaccinated by Edward Jenner), Albert Alexander (the first human treated with penicillin), Louise Brown (the first test tube baby) and Emily Whitehead (the first recipient of CAR-T cells). Now the CRISPR Journal made the unusual decision to put her on the cover.
Continue readingBetter than Casgevy? Three paths to explore
Not only sickle cell anemia, now thalassemia as well. On January 16, the Food and Drug Administration completed the approval process for the second type of hemoglobinopathy too, while the European Medicines Agency is expected to give the green light in the coming months. It took just over a decade to go from the invention of the Cas9 genetic scissors to the first approved treatment, and the excitement over the milestone achieved in record time is more than justified. Yet an article in MIT Technology Review has already turned the spotlight on the next challenges. The title is, “Vertex developed a CRISPR cure. I’ts already on the hunt for something better”. Gentler conditioning for ex vivo gene editing, new vectors for in vivo delivery, and maybe even a pill mimicking the Casgevy mechanism without modifying DNA. This is how CRISPR researchers try to out-innovate themselves.
CRISPR crops – Italy fires the starting gun
A CRISPR/Cas9-modified rice variety may be planted in a test field in northern Italy as soon as this spring after a government rule change introduced in 2023. A University of Milan team was the first research group in the country to apply for a field test under a law change that streamlines procedures for field trials of plants developed through genome editing or cisgenesis. With several other groups also planning proposals, a new wave of agricultural genetics in Italy could follow. [Please see the details in my article for Nature Italy]
CRISPeR FRENZY 