Roundup: Yearly Biotech Updates – 2018

The year 2018 has delivered many innovations and discoveries in the biotech world comprising the areas of medicine, genetics, molecular biology, stem cells, etc. Here’s a quick review of some of the notable breakthroughs and updates in 2018.

Top Biotech News For 2018

Novel Compound to Eliminate HCV

Research scientists at São Paulo State University (UNESP) in Brazil have synthesized a new compound that inhibits the replication of the hepatitis C virus in several stages of its life cycle. Such viruses have a lipid-dependent life cycle and could be affected in many ways. Using bioconjugation, the scientists conjugated Hecate—a potent bacterial inhibitor and anticancer drug—and Gallic acid, thus forming GA-Hecate. The synthetic peptide GA-Hecate proved to be a high potential antiviral that could inhibit between 50% to 99% of all major steps within the HCV infectious cycle.

Visualization of Genetic Mutation

A team of researchers from New York University and Stanford University has developed a novel fluorescent reporter that allows the detection of copy number variants (CNV) in single cells. CNV is a phenomenon in which sections of the genome are duplicated or deleted that affects a considerable number of base pairs leading to genetic diversity, driving evolutionary processes, but can also cause genetic disease and cancer. The scientists used the CNV reporter in Saccharomyces cerevisiae to study CNV formation at the GAP1 locus, which encodes the general amino acid permease, and concluded that protein fluorescence increases proportionally with gene copy number. According to the research, the molecular characterization of CNV-containing lineages shows that the CNV reporter detects different classes of CNVs, including aneuploidies, nonreciprocal translocations, tandem duplications, and complex CNVs.

CRISPR Cells Linked to Cancer Risk

Two separate studies by Sweden’s Karolinska Institute and Switzerland-based pharmaceutical company Novartis resulted that cells whose genomes are successfully edited by CRISPR-Cas9 have the potential to trigger tumors inside a patient. Karolinska researches tested CRISPR on retinal cells while the Novartis group tested on pluripotent stem cells, but they found essentially the same phenomenon. Normally, CRISPR edited cells can survive or accept the edit when they have a dysfunctional p53 protein. In such condition, when the p53 tumor suppressor protein is dysfunctional, cancer risk increases since p53 mutation is responsible for a variety of cancers: 43% colorectal cancers, 38% lung cancers, one-third of a pancreatic, stomach, and liver cancers and one-quarter of breast cancers.

Reprogrammed Stem Cells Get Green Signal in Japan

Since May 2018, the Japanese Health Ministry has given permission to scientists and doctors to treat heart patients with induced pluripotent stem (iPS) cells. These cells are produced from the revolutionary reprogramming method by inducing the cells from different body tissues such as skin and blood to revert to an embryonic-like state, which can then differentiate into other cell types. A team led by cardiac surgeon Yoshiki Sawa at Osaka University is working to regenerate damaged heart muscle using iPS cells to create a sheet of 100 million heart-muscle cells. From successful studies in pigs, they believe that grafting these sheets of cells onto a heart can improve its function.

First Monkeys Cloned with Dolly Method

Chinese scientists have created first primate clones using the same method that made the Dolly sheep two decades ago. The cloning technique used was somatic cell nuclear transfer (SCNT) that involves swapping the nucleus of a donor cell into a fertilized egg eradicated of its chromosomes. Two monkeys they cloned are named Zhong Zhong and Hua Hua. Scientists believe that genetically identical non-human primates can serve as models to study human diseases and other conditions, underlying mechanisms, and potential cures. However, many countries, including the U.S., have strict guidelines and ethical concerns about primate research.

Treating Diabetes by Regenerating Pancreatic Cells

In Mount Sinai, Egypt, a group of scientists led by Andrew F. Stewart, MD, have sequenced 38 human insulinomas—benign pancreatic tumors that secrete insulin—with 30,000 genes each and discovered the pathway that lends themselves to new drugs (to make beta cells grow and continue to make insulin). Dr. Stewart’s group is working with next-gen DNA and RNA sequencing to identify pathways to the discovery of additional beta cell regenerative drugs. So far, they have identified 86 potential candidates of such regenerative drugs that will be more easily tolerated than harmine—a similar drug to regenerate beta cells—and have reduced side effects. However, to get them in use for the clinical population would take 5-10 years.

CRISPR Genome Edited Babies

CRISPR scientist in China, He Jiankui has claimed that he has succeeded in helping create the world’s first genetically edited babies. As per his claim, twin girls were born earlier in November 2018. According to Dr. Jiankui, he has edited embryos for seven couples during fertility treatments, which has till now resulted in only one pregnancy. Jiankui used CRISPR technology to remove the CCR5 gene. Individuals without CCR5 co-receptor or homozygous carriers of CCR5 mutation are resistant to infections with the strains of HIV-1 that are most commonly encountered in sexual exposure. One of the twins had both copies of the CCR5 gene-altered while the other twin had only a single copy of the gene altered. However, the work has not been published in any journal, and many scientists reviewed the research materials provided as insufficient to draw conclusions.

Mitochondrial DNA Can be Inherited Paternally

In one of the recent studies, scientists have shown evidence on the inheritance of mitochondrial DNA from father. Usually, mtDNA is inherited from mother only in humans and most multicellular organisms. This is because the concentration of mitochondrial DNA in sperm is very low, and often the paternal mtDNA is obliterated early in the fertilization process. In the research carried out among a total of 17 individuals from three unrelated families, scientists discovered a high level of mtDNA heteroplasmy–ranging from 24% to 76%– which means mtDNA was found to be transmitted from both parents.

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