Lifeboat Foundation

Scientists have discovered that CAR T cells, traditionally used in cancer treatment, can be engineered to fight aging by eliminating senescent cells, offering a promising single-dose, lifelong treatment against aging-related diseases.

The fountain of youth has eluded explorers for ages. It turns out the magic anti-aging elixir might have been inside us all along.

Cold Spring Harbor Laboratory (CSHL) Assistant Professor Corina Amor Vegas and colleagues have discovered that T cells can be reprogrammed to fight aging, so to speak. Given the right set of genetic modifications, these white blood cells can attack another group of cells known as senescent cells. These cells are thought to be responsible for many of the diseases we grapple with later in life.

Immunizing enormous numbers of wild mice, however, is prohibitively difficult. By using genetic engineering, researchers could create white-footed mice that produced these antibodies from birth and could pass this ability on to their offspring. But did the island residents want to live with genetically engineered mice?

The answer was perhaps, but with caveats. In consulting with communities on this technology development, researchers found that community members preferred a cisgenic approach: They wanted white-footed mice that were engineered with DNA only from other white-footed mice.¹⁸ This would make the project more difficult for the researchers, and meant that a CRISPR-based gene drive, even one with limited spread, could not be used, since no white-footed mouse naturally has this gene-editing system. However, said Esvelt, “It’s their environment, so it’s their call.”

“We’re potentially causing an irreversible change to the environment,” said Telford. “We need to think about informed consent of the community as a proxy for informed consent of the environment. That’s been a real advance and something [that Esvelt] has pioneered—involving the communities from the very start.”

The CRISPR-Cas9 system has revolutionised gene-editing, but cutting DNA isn’t all it can do. From turning gene expression on and off to fluorescently tagging particular sequences, this animation explores some of the exciting possibilities of CRISPR.

Download a poster on ‘The expanding CRISPR toolbox’ here: https://www.nature.com/posters/crispr…

Continue reading “CRISPR: Gene editing and beyond” »

Researchers at Columbia University College of Dental Medicine have developed an exosome-based platform, “safeEXO-Cas,” that significantly enhances the delivery of CRISPR/Cas9 genome editing components to specific cells and tissues.

New rules on NGTs — here’s what it means.

During her chemistry Nobel Prize lecture in 2018, Frances Arnold said, “Today we can for all practical purposes read, write and edit any sequence of DNA, but we cannot compose it.” That isn’t true anymore.

Advances in gene editing technology have led to the first successful transplant of a pig kidney into a human.

Joint research led by Yutaro Shuto, Ryoya Nakagawa, and Osamu Nureki of the University of Tokyo determined the spatial structure of various processes of a novel gene-editing tool called “prime editor.” Functional analysis based on these structures also revealed how a “prime editor” could achieve reverse transcription, synthesizing DNA from RNA, without “cutting” both strands of the double helix. Clarifying these molecular mechanisms contributes greatly to designing gene-editing tools accurate enough for gene therapy treatments. The findings were published in the journal Nature.

The 2020 Nobel Prize in Chemistry was awarded to Jennifer Doudna and Emmanuelle Charpentier for developing a groundbreaking yet simple way to edit DNA, the “blueprint” of living organisms. While their discovery opened new avenues for research, the accuracy of the method and safety concerns about “cutting” both strands of DNA limited its use for gene therapy treatments. As such, research has been underway to develop tools that do not have these drawbacks.

The prime editing system is one such tool, a molecule complex consisting of two components. One component is the prime editor, which combines a SpCas9 protein, used in the first CRISPR-Cas gene editing technology, and a reverse transcriptase, an enzyme that transcribes RNA into DNA. The second component is the prime editing guide RNA (pegRNA), a modified guide RNA that identifies the target sequence within the DNA and encodes the desired edit. In this complex, the prime editor works like a “word processor,” accurately replacing genomic information. The tool has already been successfully implemented in living cells of organisms such as plants, zebrafish, and mice. However, precisely how this molecule complex executes each step of the editing process has not been clear, mostly due to a lack of information on its spatial structure.

It’s estimated that almost half of the world’s population — about 3.7 billion people under the age of 50 — are infected with (HSV-1), which can cause oral herpes. About half a billion people between the ages of 15 and 49 are infected with herpes simplex virus-2 (HSV-2), the cause of genital herpes. There are therapeutics that can eliminate some symptoms of herpes, like blisters, but there is no cure for the infection, and those who are infected can spread the virus to others. Studies have suggested that HSV-1 may increase the risk of dementia, and HSV-2 raises the risk of HIV infection.

Scientists have now developed a gene therapy that can eliminate as much as 90 percent of oral herpes and 97 percent of genital herpes infections in pre-clinical mouse models. The gene therapy also reduced the level of virus that was released from an individual in a mouse model of the infection. These reductions took about one month to be completed, and more of the virus seemed to be eliminated over time. The work has been reported in Nature Communications.