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Lipid Nanoparticles Deliver CRISPR/Cas9 into Organs with High Efficiency

Researchers at Tufts University and the Chinese Academy of Sciences have developed a new lipid nanoparticle which can deliver CRISPR/Cas9 gene editing tools into organs with high efficiency, suggesting that the system is promising for clinical applications.

The CRISPR/Cas9 system is currently being investigated as a way to treat a variety of diseases with a genetic basis, including Duchenne muscular dystrophy, Huntington’s, and sickle cell disease. While the system has significant promise, there are some issues that need to be resolved before it can be used clinically. CRISPR/Cas9 is a large complex, and it is difficult to get it inside cell nuclei where it is needed for gene editing.

Scientists have tried a variety of delivery vehicles for CRISPR/Cas, which are intended to carry the gene editing tools to their location and help them enter the cell and nucleus. These have included viruses and various types of nanoparticle. However, to date, these have suffered from low efficiency, whereby very little of the delivered agent reaches the cells or organs where it is needed.

Researchers create ‘epigenetic atlas’, heralding leap forward in disease diagnosis

This atlas of human CoRSIVs,” they write, “provides a resource for future population-based investigations into how interindividual epigenetic variation modulates risk of disease,” and may well transform understanding of the causes of illness in the human body.


A project 370 times larger than the Human Genome Project bears first fruit. Stephen Fleischfresser reports.

A Common Cold Virus Wiped Away Bladder Cancer in One Patient

A simple cold virus could wipe out tumors in a form of bladder cancer, a small new study suggests.

Though the idea of using viruses to fight cancer isn’t new, this is the first time a cold virus effectively treated an early-stage form of bladder cancer. In one patient, it eliminated a cancerous tumor, the group reported July 4 in the journal Clinical Cancer Research.

A group of researchers conducted an early-stage clinical trial in which they infected 15 bladder cancer patients with coxsackievirus A21, which is one of the viruses that cause the common cold. Coxsackievirus is not a genetically modified virus; it’s “something that occurs in nature,” said senior author Hardev Pandha, a professor of medical oncology at the University of Surrey in England. [Exercise May Reduce the Risk of These 13 Cancers].

A Link Between Telomere Shortening and Species Aging

A recently released study from Maria Blasco and her team of researchers at the Spanish National Cancer Research Center (CNIO) shows that the rate of telomere shortening is strongly correlated with the maximum lifespan of animal species.

Telomeres

Telomeres, which are simply repeating segments of DNA on the ends of our chromosomes, serve two critical functions: They protect the ends of our chromosomes, preventing genetic damage, and they serve as a clock, limiting the number of times that our cells can divide. This limit, known as the Hayflick limit, serves as a basic defense against cancer. However, telomere attrition is a primary hallmark of aging and leads to cellular senescence and other age-related disorders.

Decoding the Axolotl genome

A team of researchers led by scientists in Vienna, Dresden and Heidelberg has decoded the entire genetic information of the Mexican salamander axolotl. The axolotl genome, which is the largest genome ever to be sequenced, will be a powerful tool to study the molecular basis for regrowing limbs and other forms of regeneration.

Salamanders have long served as valuable biological models for developmental, regeneration and evolutionary studies. In particular, the Mexican axolotl Ambystoma mexicanum has received special attention due to its astounding ability to regenerate body-parts. If the cannibalistically inclined animal loses a limb, it will regrow a perfect substitute within weeks, complete with bones, muscles and nerves in the right places. Even more fascinating, the axolotl can repair severed spinal cord and retinal tissue. These qualities and the relative ease in breeding have made it a favourite biological model, cultivated in the lab for more than 150 years.

Self-destructing mosquitoes and sterilized rodents: the promise of gene drives

The technical challenges are not as daunting as the social and diplomatic ones, says bioengineer Kevin Esvelt at the Massachusetts Institute of Technology (MIT) Media Lab in Cambridge, who was among the first to build a CRISPR-based gene drive. “Technologies like this have real-world consequences for people’s lives that can be nearly immediate.”


Altering the genomes of entire animal populations could help to defeat disease and control pests, but researchers worry about the consequences of unleashing this new technology.

Scientists successfully edit a long-locked part of plant DNA, improving crop security

Think of DNA and chances are the double helix structure comes to mind, but that’s only one piece of the puzzle. Another major part is mitochondrial DNA, and in plants that’s even more important – and so complex that scientists haven’t yet been able to edit the genes in there. Now a team of Japanese researchers has managed to do just that, which could help improve the genetic diversity of crops.

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