Aurora magazine

The first phase of a clinical trial for a new gene therapy against Type B hemophilia is underway. Researchers have also recruited 5 new patients to be given a new version of the therapy. For the time being, the researchers used SPK-9001 on only one patient, who is around the 35th week of follow-up. According to the first data, however, the results obtained from the new process are comparable to those obtained with the old one.

Gene therapy SPK-9001 should help those with type B hemophilia to produce factor IX. For the time being, neither the first 10 participants nor what they added at a later date showed particular side effects. They have also stopped concentrate IX concentrate infusions, which also makes good hope for efficacy.

All patients showed an increase in factor IX levels, making infusions of the same high. Data on the 7 patients who completed a one-year follow-up show a lasting efficacy of therapy. None of them has shown any cases of bleeding, even without the use of preventative prophylaxis. Everyone has reported an improvement in the quality of life, which is good for the future.

The new version of SPK-9001 will be used in phase 3 of the clinical trial. If this stage is fine, gene therapy will be ready for approval and subsequent marketing.

Source: hemophilianewstoday.com

The Bambino Gesù Hospital in Rome has been the stage for the incredible separation of two Algerian siamese twins. The operation lasted 10 hours and involved a team of 40 people. The Roman hospital is preparing for a new challenge. At the end of November, doctors will shed two Burundi sisters.

The new separation of Siamese is anticipated even more complicated than the previous one. The girls are two and a half months old and are tied to the lowest point of the back. They are attached to the legs, the spinal cord, the bladder and the rectum, sharing a single anus. This last point makes the operation even harder: physicians will have to create a further anus, to give the two girls the same life expectancy.

The news of separation action on Algerian girls has increased the optimism of the operation. But doctors keep us putting pallets: science has its limits and the conditions of Burundi's twins is not easy. In some cases it is impossible to separate a pair of siams without killing one or both of them. There is only one hope that the new case is not one of these.

Although doctors do not bother with the outcome of the future operation, there is good news about the past. One month after the intervention, the little Rayenne and Djihene are fine. They have crossed and traverse difficult times, but the hospital seems normal and devoid of surprises.

Source: ansa.it

The Zika virus uses Troy's horse strategy: first attacks the immune cells, then uses them to spread to the fetal brain. The discovery comes from researchers at the University of California, San Diego, and their Brazilian counterparts.

During the development of the embryo, the microglia cells developed into the veal sac, which then gave rise to macrophages. Their job is to keep the body clean, eliminating harmful agents and damaged cells. The Zika virus starts the infection right from the microglia cells. When they spread to the central nervous system, they carry the virus with it and facilitate its spread.

As it is now known, the Zika virus is harmless to adults and devastating to fetuses. It attacks the developing brain, killing neural stem cells and causing microcephaly. However, it was unclear how the virus was transmitted and spread in the fetal brain. Apparently the answer is in the newborn immune system, used as a troika horse from Zika.

To test their hypothesis, researchers used pluripotent stem cells induced. They have created microglia cells and neural progenitor cells, both of which are essential to fetal development. They then imitated the way the two types of cells interact and exposed them to the virus. The microglia cells immediately swallowed the Zika virus to eliminate it. However, the virus remained active and used microglia cells to reach progenitor cells.

The team tested a drug called Sofosbuvir, used against hepatitis C. The drug limits viral infection and decreases the number of dead stem cells. Additional tests will be needed, but the first results are encouraging.

Source: medicalxpress.com

Two Broad Institute and Harvard University teams have developed new extensions for CRISPR. The two techniques allow you to rewrite single letters or DNA bases and RNA.

The CRISPR genetic editing technique cuts a section of the double helix of the DNA, replaced by a healthy version. It's an effective but somewhat violent approach to DNA. Extensions, on the other hand, allow you to act in a more targeted manner. Instead of cutting the double helix, they use enzymes to change the order of bases that make up DNA and RNA. This way you get a new base without acting on the surrounding ones.

Findings are particularly relevant for genetic diseases with point mutations. They are illnesses caused by changes in single nucleotides, where a single base is put in the wrong place.

The technique developed by David Liu acts on the layout of the adenine atoms and turns them into guanine. In this way, the pair of A-T bases becomes a C-G pair. The procedure proved to be effective in 50% of cases and did not lead to collateral changes.

The second technique, discovered by the Feng Zhang team, acts on the RNA. The researchers turned the adenine into iosin. The components that produce cellular proteins read iosin as guanine. The method allows you to make a temporary change only because it acts on the RNA and not on the DNA. This means that you can repeat the process several times, even by making improvements.

Source: focus.it