Europe is about to launch its first CRISPR-based human genetic modification trial

Release date: 2018-04-19

Human genetic modification trials will also be conducted in Europe following regulatory approval of DNA splicing therapy.

Scientists at CRISPR Biotech hope to adopt new therapies for a devastating blood disease (beta thalassemia).

China is the first country to use CRISPR to cure beta thalassemia. It is also the disease. Now, Europe will also take a tentative first step. "Nature" headlines: Research groups including Chinese scholars use CRISPR to successfully implement human embryo editing

Professor Robin Lovell-Badge, research leader at the Francis Crick Institute in London, said: "One day, when we look back today, this will be a new beginning for gene therapy."

Haemoglobin carries the oxygen needed by the body. If the cells do not get enough oxygen, patients with such defects will develop symptoms such as bone deformity, anemia, slow growth, fatigue and shortness of breath. Scientists hope to use the Cas9 enzyme to cut off the mutant gene and restore normal hemoglobin levels.

Researchers have extensively tested the cutting efficacy of the system, and look forward to the day when the therapy will also apply to humans. Scientists plan to extract stem cells from the human body, let them grow in the laboratory, and then increase fetal hemoglobin production. Although these proteins can replace adult hemoglobin, fetal hemoglobin production is usually inhibited after humans enter adulthood.

When the gene that inhibits fetal hemoglobin is removed, the patient's bone marrow can again produce high levels of hemoglobin.

Darren Griffth, a professor of genetics at the University of Kent, told an interview with the Sunday Telegraph: "Everything I have seen shows that this method is very safe and effective. I think the (test) results will be positive."

The University of Pennsylvania has begun recruiting cancer patients who have undergone therapeutic trials using CRISPR.

Source: Biopass

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