Pulse 125: Prime Editing May Cure Most Genetic Diseases

28 October 2019
Giulio Prisco

DNA Prime Editing

The discovery of a new precise, efficient, and highly versatile gene editing technique, which could be used to correct up to 89 percent of known genetic diseases, is all over the press.

"A major aspiration in the molecular life sciences is the ability to precisely make any change to the genome in any location. We think prime editing brings us closer to that goal," said senior scientist David Liu in a press release. "With prime editing, we can now directly correct the sickle-cell anemia mutation back to the normal sequence and remove the four extra DNA bases that cause Tay Sachs disease ..."

"Prime editing is the beginning," added Liu as reported by BBC News, "rather than the end of a long-standing aspiration in the molecular life-sciences to be able to make any DNA change in any position of a living cell or organism, including potentially human patients with genetic diseases."

"Prime editing offers a new way to make changes to DNA while avoiding some of the drawbacks and clunkiness of traditional CRISPR," notes Smithsonian Magazine. "Where the familiar CRISPR technique fully cleaves a strand of DNA in two, often creating some tiny, inadvertent genetic changes as byproducts, prime editing begins by slicing just one of the two strands of the double helix. The method is sleeker, less invasive, and offers the potential for precision genetic editing."

It seems likely that, when perfected and made available for clinical trials and operational therapies, prime editing could be a godsend for those who suffer from genetic diseases.

Of course, some people are only able to complain. "But should Crispr also be used to 'correct' deafness, for example, and by extension, eradicate a rich and vibrant deaf community?" asks The Guardian. This question is worthy skepticism, I think.

BBC News notes that the big challenge for gene editing therapies is "getting the molecular machinery that is capable of performing these edits into the right parts of the human body and to ensure they are safe." Motley Fool agrees.

Prime Gene Editing May Correct Most Genetic Diseases

Scientists at Broad Institute of MIT and Harvard University have developed a new CRISPR genome-editing approach, called “prime editing.” It is capable of directly editing human cells in a precise, efficient, and highly versatile way.

A research paper published in Nature describes prime editing’s ability to precisely correct gene variants that cause two diseases. To correct sickle-cell anemia, it requires the conversion of a specific T to an A. To correct Tay Sachs disease, it requires the removal of four DNA letters at a precise location in the genome.

Prime editing is based on combining CRISPR-Cas9 and a reverse transcriptase. The scientists are persuaded that prime editing has the potential to correct up to 89 percent of known disease-causing genetic variations.

Anti-Cancer Effects of Drug Derived from Turmeric

Researchers at Nara Institute of Science and Technology have found out how an experimental drug agent stops cancer cells from growing. Pentagamavumon-1 (PGV-1), an analogue of a molecule found in turmeric, was already indicated as possibly having anti-cancer effects.

In a new study published in Scientific Reports, tests on cancer cells and animals reveal that these anti-cancer effects come from PGV-1 inhibiting a series of enzymes responsible for the metabolism of reactive oxygen species.

This finding is expected to clarify how modifications to PGV-1 will lead to its use for cancer treatment.

Toward More Effective Cancer Immunotherapy

Scientists at Washington University have found a way for cancer immunotherapy to spur a more robust immune response.

Much immunotherapy for cancer is designed to prompt immune cells called killer T cells to attack the cancer cells. But a research paper published in Nature suggests recruiting other T cells, called helper T cells. With the helper cells, the immune system is more likely to respond to fight cancer.

The research results could lead to the development of better cancer vaccines and more effective immunotherapy drugs called checkpoint inhibitors.

Nanotechnology Delivers Drugs to Cancer Cells

Researchers at Washington State University and Pacific Northwest National Laboratory have developed a precise and non-toxic treatment that targets lung cancer cells at the nanoscale. It is able to effectively kill the cells even at a low dose.

The researchers used biologically-inspired nanotubes to deliver cancer-killing drugs in a targeted manner.

As reported in a study published in Small, the researchers tested the nanotubes on lung cancer cells and found that they delivered the chemotherapy drug doxorubicin directly into the fast-dividing cancer cells. It resulted in highly efficient cancer killing while using less chemotherapy drugs.

Bioprinting Living Cells and Tissues

Scientists at Vienna University of Technology have developed a high-resolution bioprinting process.

The process permits embedding cells in a 3D matrix, printed with micrometer precision, at a printing speed of one meter per second. This is orders of magnitude faster than previously possible.

Tissue growth and the behavior of cells can be controlled and investigated particularly well by embedding the cells in a delicate 3D framework.

The new bioprinting technique is described in a research paper published in Advanced Healthcare Materials. It could enable production of tailor-made tissues using stem cells.

Radiotherapy for Pancreatic Cancer

Researchers at Osaka University and University of Heidelberg have tested a new radiotherapy technique. It sends alpha-emitting particles to stroma cells in pancreatic tumors.

The method is described in a study published in Journal of Nuclear Medicine.

It slowed tumor growth in laboratory mice with minimal side effects. This points to a potential treatment option for patients with pancreatic cancer.

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