Tag Archives: genes

CRISPR-SKIP, New Gene Editing Technique

What if doctors could treat previously incurable genetic diseases caused by errors or mutations in genes? Thanks to new research by American scientists at the University of Illinois, we are one step closer to making that a reality. Published in Genome Biology, their work is based on CRISPR-Cas9, a groundbreaking genome editing system.

Typically, cells in the body “readDNA to produce the proteins needed for different biological functions. . Scientists can change how the DNA is read using CRISPR gene-editing technology. CRISPR-Cas9 is often used to cut out specific areas of DNA and repair faulty genes. In the current study, the researchers modified existing technology to create CRISPR-SKIP. Instead of breaking DNA to cut faulty genes out, CRISPR-SKIP changes a single base of the targeted DNA sequence, causing the cell to skip reading that section of DNA.

According to the study authors, CRISPR-SKIP can eliminate faulty sections of DNA permanently, allowing for long-lasting treatment of some genetic diseases with one treatment. They successfully tested their technique in cell lines from both mice and humans. The scientists aim to test the method in live organisms in the future.

CRISPR-SKIP has the potential to help treat many diseases such as cancer, rheumatoid arthritis, Huntington’s disease, and Duchenne muscular dystrophy to name a few. Because the method only requires editing of a single base, it is simple, precise, and adaptable to a variety of cell types and applications.

Source: https://news.illinois.edu/
AND
https://www.medicalnewsbulletin.com/

Genes Behind Humankind’s Big Brain

Scientists have pinpointed three genes that may have played a pivotal role in an important milestone in human evolution: the striking increase in brain size that facilitated cognitive advances that helped define what it means to be human. These genes, found only in people, appeared between 3 and 4 million years ago, just prior to a period when the fossil record demonstrates a dramatic brain enlargement in ancestral species in the human lineage, researchers said. The three nearly identical genes, as well as a fourth nonfunctional one, are called NOTCH2NL genes, arising from a gene family dating back hundreds of millions of years and heavily involved in embryonic development. 

The NOTCH2NL genes are particularly active in the reservoir of neural stem cells of the cerebral cortex, the brain’s outer layer responsible for the highest mental functions such as cognition, language, memory, reasoning and consciousness. The genes were found to delay development of cortical stem cells into neurons in the embryo, leading to the production of a higher number of mature nerve cells in this brain region.

The cerebral cortex defines to a large extent what we are as a species and who we are as individuals. Understanding how it emerged in evolution is a fascinating question, touching at the basic origins of mankind,” said developmental neurobiologist Pierre Vanderhaeghen of Université Libre de Bruxelles and VIB/KULeuven in Belgium.

It is the ultimate evolutionary question and it is thrilling to work in this area of research,” added biomolecular engineer David Haussler, scientific director of the University of California, Santa Cruz Genomics Institute and a Howard Hughes Medical Institute investigator.

Source: https://www.reuters.com/