CRISPR | Dispatches From The Front

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CRISPR ‘will provide cures for genetic diseases that were incurable before,’ says renowned biochemist Virginijus Šikšnys | Live Science

Posted on February 20, 2024 by knjohn | Leave a comment

Scientists introduced CRISPR to the world as a gene-editing tool in the summer 2012, when landmark papers from two independent groups demonstrated how the system could be wielded to make cuts in DNA. Now, less than 12 years later, we’re seeing CRISPR put to use in groundbreaking medical treatments.

Virginijus Šikšnys was a senior author of one of those paradigm-shifting papers.

“It’s really rewarding to see how fast the fundamental discoveries that were made in the lab actually are translated into the clinics,” said Šikšnys, who is chief scientist and head of the Department of Protein-DNA Interactions at the Vilnius University Institute of Biotechnology in Lithuania.

CRISPR used to ‘reprogram’ cancer cells into healthy muscle in the lab | Live Science

Posted on September 5, 2023 by knjohn | Leave a comment

Scientists have transformed cancer cells into healthy muscle tissue in the lab using CRISPR gene-editing technology — and they hope new cancer treatments can be built on the back of this experiment.

In a study published Aug. 28 in the journal PNAS, researchers found that disabling a particular protein complex in cells of rhabdomyosarcoma (RMS) — a rare cancer in skeletal muscle tissue that mainly affects children under age 10 — in the laboratory causes the tumor cells to turn into healthy muscle cells.

Although the research is still in its early days, this process of “resetting” cancer cells into healthy cells, broadly known as differentiation therapy, has already been tested in other types of cancer, such as bone and blood cancer. Four drugs have been approved by the U.S. Food and Drug Administration (FDA) to treat the latter disease and generally work by inhibiting a specific protein in the cancer cells.