Lifeboat Foundation

Down syndrome (DS) is one of the most prevalent genetic disorders in humans. The use of new approaches in genetic engineering and nanotechnology methods in combination with natural cellular phenomenon can modify the disease in affected people. We consider two CRISPR/Cas9 systems to cut a specific region from short arm of the chromosome 21 (Chr21) and replace it with a novel designed DNA construct, containing the essential genes in chromatin remodeling for inactivating of an extra Chr21. This requires mimicking of the natural cellular pattern for inactivation of the extra X chromosome in females. By means of controlled dosage of an appropriate Nano-carrier (a surface engineered Poly D, L-lactide-co-glycolide (PLGA) for integrating the relevant construct in Trisomy21 brain cell culture media and then in DS mouse model, we would be able to evaluate the modification and the reduction of the active extra Chr21 and in turn reduce substantial adverse effects of the disease, like intellectual disabilities. The hypothesis and study seek new insights in Down syndrome modification.

Keywords: Down syndrome, CRISPR/Cas9, Designed DNA construct, Poly D L-lactide-co-glycolide (PLGA), Nano-carrier, Chromosome 21 inactivation.

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CRISPR gene editing leads to improvements in vision for people with inherited blindness, a recent clinical trial shows.

“Engineering the brain”. There was no intelligent design, and as a result, body organs do not resemble machines. Once we start building machines like body organs — with utility functions, self-organization and cells as building blocks, we can mesh engineering and evolutionary principles to arrive at better organisms.

Each CRISPR system has two parts: a strand of RNA that matches the target and a protein that makes the edit. The most commonly used protein for gene editing is called “Cas9,” but scientists have discovered CRISPRs with other proteins that give them unique capabilities — while CRISPR-Cas9 slices through DNA, for example, CRISPR-Cas13 targets RNA.

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The results of a metagenomic study from the University of Trento suggest that the CRISPR toolbox will need to make room for another CRISPR enzyme. The disruption should be minimal because the newly identified enzyme is unusually compact. It consists of just over 1,000 amino acids. And yet it is also strongly active and highly precise. The hope is that it can be packaged with guide RNA within the tight quarters afforded by adeno-associated virus (AAV) vectors, and thereby expand the use of in vivo gene editing in therapeutic applications.

The study was led by Anna Cereseto, PhD, and Nicola Segata, PhD, of the department of cellular, computational, and integrative biology. Cereseto leads a laboratory that develops advanced genome editing technologies and their application in the medical sector. Segata is the head of a laboratory of metagenomics, where he studies the variety and characteristics of the human microbiome and its role in health. Their collaboration has led to the identification, in a bacterium of the intestine, of new CRISPR-Cas9 molecules that could have a clinical potential to treat genetic diseases.

Detailed findings from the study recently appeared in Nature Communications, in an article titled, “CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing.”

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