Optimized nano-micelles can induce efficient genome editing in the mouse brain
Mar 12 2021
The research group of Deputy Principal Research Scientist Dr. Satoshi Uchida (Associate Professor, Kyoto Prefectural University of Medicine) at the Innovation Center of NanoMedicine (Director General: Prof. Kazunori Kataoka, Location: Kawasaki-Japan, Abbreviation: iCONM) reported that optimized nano-micelles can induce efficient genome editing in the mouse brain.
The 2020 Chemistry Nobel Prize-winning technology CRISPR/Cas9 holds great promise for treating various diseases such as congenital disorders and viral infections, by correcting the disease-specific genomic sequences.
This technology attracted increasing attention in the last decade as it allows easy targeting of genomic sequences with high precision and low level of undesired off-target gene editing. However, safe and efficient delivery of the Cas9 DNA cutting enzyme and guide RNA (gRNA), which is a short piece of RNA that guides C
Researchers in China and Australia have reported the discovery of novel bat coronaviruses that further reveal the diversity and complex evolutionary history of these viruses.
Whole genome sequencing may guide blood cancer treatment
For certain blood cancers, such as acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), deciding whether patients need an aggressive treatment typically hinges on a set of lab tests to identify genetic changes.
Some of these tests rely on technology that was invented more than 60 years ago and has been used clinically for the past three decades.
Now, a new study from Washington University School of Medicine in St. Louis shows that whole genome sequencing is at least as accurate and often better than conventional genetic tests that help determine the treatment for a patient s blood cancer. Genome sequencing technology continuously is decreasing in cost and recently reached a level similar to that of conventional testing. In addition, results can be returned to patients in just a few days, making whole genome sequencing a potentially viable approach for determining the best treatment regimen for a particular patien
Novel CRISPR-based gene editor developed to correct mutations that cause genetic disorders
A team of researchers from the Agency for Science, Technology and Research s (A STAR) Genome Institute of Singapore (GIS) have developed a CRISPR-based gene editor, C-to-G Base Editor (CGBE), to correct mutations that cause genetic disorders. Their research was published in
Nature Communications on 2 March 2021.
One in seventeen people in the world suffers from some type of genetic disorder. Chances are, you or someone you know - a relative, friend, or colleague - is one of approximately 450 million people affected worldwide. Mutations responsible for these disorders can be caused by multiple mutagens - from sunlight to spontaneous errors in your cells. The most common mutation by far is the single-based substitution, in which a single-base in the DNA (such as G) is replaced by another base (such as C). Countless cystic fibrosis patients worldwide have C instead of G, leading to defective p