Aurora magazine

Texas University researchers used CRISPR to correct the mutation due to Duchenne's dystrophy. For the time being they have performed on human cardiomyocytes and on animal models. Both cultured cell cultures and cavities recovered part of their functions. This demonstrates the potential of CRISPR for the development of new treatments, as well as for the prevention of diseases.

A 2014 study by the same authors used CRISPR-Cas9 to correct mice in a key gene for the disease. In the present study they applied a similar strategy, but using CRISPR-Cpf1. The latter is simpler and allows you to correct mutations that are not reachable before. In this way you will be able to act on a broad spectrum of issues with better results.

The team converted the fibroblasts of patients with Duchenne into pluripotent stem cells induced. It then deleted the mutations linked to the production of dystrophin, a key protein in the development of the disease. Subsequently, they developed RNA guidance that could restore the correct protein functions. They also induced a cellular respiration rate closer to that of a healthy heart.

For the in vivo part of the tests, the researchers injected the Cpf1 endonucleases together with the RNA guide and a single strand of a DNA sequence. In this way they facilitated homologous recombination, thus correcting the mutation in mice zygotes. They then implanted the zygotes in some "surrogate mothers" and on 24 puppies 5 they had the correct version of the gene.

Source: the-scientist.com

The "Sergio Bonelli Center for Congenital Renal and Urinary Malformations" was inaugurated: prevention and care of kidney damage from the fetus to the baby ". The center has opened at the Policlinico di Milano and aims to study kidney damage caused by congenital malformations. In this way it is hoped to develop new strategies to fight certain pathologies already from the uterus.

Congenital renal damage is the first cause of dialysis and kidney transplantation in pediatric age. It develops in the early months of pregnancy, during the formation of the organs. Causes can be genetic abnormalities or urinary flow obstructions that result in damage to kidney development. Many of these obstacles are evident in normal prenatal screening. To reduce permanent damage, however, is necessary. Beyond health repercussions, this kind of harm has heavy psychological and social implications on children.

Among the projects of the center is the Studio PROFET, a multicentric European study aimed at identifying possible kidney damage from obstruction in the fetus. The aim is to develop new prenatal screening tests to carry out a diagnosis of fetal urine. This way you will know when to resort to new technology, designed to remove the obstruction. In addition to PROFET there is PREDICT, which aims to evaluate the efficacy of antibiotic prophylaxis on infants with renal abnormalities. The study has already involved 153 children.

Source: observatoriomalattierare.it

The University of Miami is about to launch the first trial for the Zika virus vaccine. It is one of the first DNA vaccines against the virus promoted by the National Institutes of Health. Since Miami is a high-risk area of ​​pandemic, experimentation in the area will be crucial. The first goals are to determine the safety and dosage of the vaccine.

The first phase involves recruiting individuals from Miami who will receive several vaccine doses to test their safety. The participants will be 90 men and women between the ages of 18 and 35. Women should not be pregnant. The second phase of trials will serve to determine the efficacy of the vaccine. It will involve 2,400 individuals never infected with the Zika virus, who live in areas with high risk of contagion. Half of them will receive the vaccine, half the placebo.

NIAID's Vaccine Research Center (VRC) researchers have developed a DNA vaccine. The strategy is the same as that used for the West Nile fever vaccine. The vaccine contains a double-helix DNA molecule, within which there are genes encoding two viral proteins. Once injected into the muscle, the proteins create false Zika viruses, which trigger the immune response of the body. As the infectious material is completely missing, the risk of infection is null and total protection should be obtained.

The trials have a wide-ranging study on animal models that the vaccine would be safe and able to neutralize the virus. However, it is necessary to ascertain whether this also applies to humans. Phase Two participants will be followed for almost 2 years, so as to check for any possible infection or adverse reactions.

Source: miami.edu

The zebrafish is extremely prolific: the female produces an average of 200 eggs a week. Embryos are transparent and develop rapidly within 2-3 months.
The biological mechanisms of the embryonic growth of Danio rerio resemble many aspects to human beings. For these reasons and for the ease with which researchers succeed in inducing mutations in their DNA, zebrafish has been chosen as a genetic model for the study of diseases and defects in human development.

By analyzing the interactions between fish genes, experts hope to shed light on the regulatory mechanisms of gene expression in human DNA.
Dozens of research centers around the world have specialized in zebra fisheries over the last few years and the competition between Europe and the United States in this area is very strong, both purely scientific and from the point of view of 'Pharmaceutical and biotechnology industry.

The ZF-MODELS project

The objective of the European Commission and the Swiss National Fund for Scientific Research is to support the competitive effort of the old continent.
This is how the ZF-MODELS project, which was launched in January and coordinated by the Max Planck Institute in Tübingen, was attended by fifteen research institutes in France, Germany, Great Britain, Italy, Norway, the Netherlands and Switzerland with a Politecnico Zurich. The European Commission will contribute to the total cost of the initiative with twelve million euros, the Swiss Fund with another 400,000 euros.

The most modern techniques of DNA manipulation and analysis will be applied massively: researchers will produce thousands of mutant fish to study the effects of mutations, determine the function of each gene, and correlate the interactions between them.

All the information gathered within the project will come together in anatomical and genetic zebrafish atlas that will be available free of charge to the scientific community and the public interested on the zf-models site.

The zebrafish in Zurich

The Zurich Politecnico research group taking part in the ZF-MODELS project, led by the neurobiologist Stephan Neuhaus, has long been using zebrafish as a model for embryonic development analysis. "Our specific field of interest is the development of the visual apparatus," explains Neuhaus, "in practice we produce and analyze genetic mutations that involve visual impairment."

The Swiss neurobiologist and his team have developed techniques to detect blind larvae. "Let's investigate the causes of the blindness of these mutated specimens," says Neuhaus, "and identify the molecular defects at the origin of visual apparatus malfunction."

Some of the mutations have similarities with hereditary human diseases, such as retinal dystrophy. "We therefore hope that our work will be useful in the medical field."