Shulou(Shulou.com)11/24 Report--

Thanks to CTOnews.com netizens for the clues of star-picking delivery! CTOnews.com, December 13 (Xinhua)-- British scientists have treated a 13-year-old patient with T-cell leukemia using a revolutionary new gene therapy, base editing (base-edited), the Guardian reported on December 11, the world's first case of this technique.

University College London Greater Ormond Street Children's Hospital said the girl had previously failed to respond to other treatments, but had no cancer cells detected after receiving base editing treatment in May this year. However, doctors also said that it remains to be seen whether she will no longer have related symptoms in the future.

Waseem Qasim, a researcher at University College London who worked on the project, said: "this is our most complex cell engineering to date, paving the way for other new treatments and eventually leading to a better future for sick children."

The technology behind this innovative new therapy has been around for less than five years, and researchers initially described it as "CRISPR 2.0." unlike previous CRISPR gene editing techniques, which are basically like cut and paste, base editing is more targeted and accurate.

Our human genome consists of 3 billion base pairs made up of the letters A, C, G and T, and base editing allows single-letter editing of genes without causing DNA fragmentation. If CRISPR's previous iterations were like molecular scissors, base editing was like using pencils and erasers-changing a single letter in a base pair to change a particular cellular mechanism.

T-cell leukemia is a cancer caused by a defect of white blood cells called T cells. These white blood cells do not develop normally and grow too fast, interfering with the growth of blood cells in the body. Standard treatment includes bone marrow transplant and chemotherapy, but in Alisha's case, these traditional treatments have failed to stop the disease, and her only option seems to be palliative care.

In the past few years, scientists have made extraordinary breakthroughs in the gene therapy of cancer. Genetic editing of patients'T cells to target specific cancers is not a particularly new technique. However, the challenge for T-cell acute lymphoblastic leukemia is that genetically edited T cells target other T cells, often resulting in modified cells destroying other modified T cells.

As a result, new base editing techniques enable researchers to add several new modifications to T cells, which are provided by healthy donors. Base editing changes several key markers that recognize immune cells as T cells. This means that the edited cells are basically invisible to other T cells.

Other base editors of T cells have deleted donor-specific markers, turning the cells into a "universal" treatment. Crucially, this means that the treatment can become an off-the-shelf drug available to many patients, in contrast to the current slow and expensive personalized nature of T-cell therapy.

Simon Waddington, a professor of gene therapy at University College London, said previous gene therapy had had some astonishing successes, but the tedious process of creating treatments limited its wide range of feasibility. Collecting immune cells from patients, genetically modifying these specific cells, and then transplanting them back into the patient is a slow and time-consuming process.

"in this latest study, there is a cell bank-a single cell bank that can be used to treat many patients," Waddington said. "this enables the scalability, commercial viability and standardization of pharmaceutical products."

Within a month of receiving experimental base editing therapy, Alisha was completely freed from leukemia. Six months later, Alisha is still in complete remission.

CTOnews.com learned that the preliminary clinical trial hopes to recruit 10 more patients in the next few years, and that this kind of leukemia treatment is just the tip of the iceberg of base editing. At least three more trials are under way to test base editing for sickle cell anaemia, high cholesterol and a blood disease called β-thalassemia.

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