She worked in top labs at Stanford and Harvard. Now she wants to disrupt a 100-Billion-Dollar market by rejuvenating your skin using genetic engineering.
Category: bioengineering – Page 71
The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now trying to figure out what killed him.
Terry Horgan, a 27-year-old who had Duchenne muscular dystrophy, died last month, according to Cure Rare Disease, a Connecticut-based nonprofit founded by his brother, Rich, to try and save him from the fatal condition.
Although little is known about how he died, his death occurred during one of the first studies to test a gene editing treatment built for one person. It’s raising questions about the overall prospect of such therapies, which have buoyed hopes among many families facing rare and devastating diseases.
As nanotechology burrows into an increasing number of medical technologies, new developments in nanoparticles point to the ways that treatments can today be nanotechnologically targeted. In one case, would-be end effectors on microrobots are aimed at clearing up cases of bacterial pneumonia. In another, a smart-targeting system may decrease clotting risks in dangerous cases of thrombosis.
Scientists from the University of California, San Diego, demonstrated antibiotic-filled nanoparticles that hitch a ride on microbots made of algae to deliver targeted therapeutics. Their paper was recently published in Nature Materials. As a proof of concept, the researchers administered antibiotic-laden microbots to mice infected with a potentially fatal variety of pneumonia (a strain that is common in human patients who are receiving mechanical ventilation in intensive-care settings). All infections in the treated mice cleared up within a week, while untreated mice died within three days.
The algae–nanoparticle hybrid microbots were effectively distributed to infected tissue through lung fluid and showed negligible toxicity. “Our goal is to do targeted drug delivery into more challenging parts of the body, like the lungs,” said bioengineering professor Liangfang Zhang in a press statement. “And we want to do it in a way that is safe, easy, biocompatible, and long lasting.”
Genetic engineering is a rapidly progressing scientific discipline, with tremendous current application and future potential. It’s a bit dizzying for a science communicator who is not directly involved in genetics research to keep up. I do have some graduate level training in genetics so at least I understand the language enough to try to translate the latest research for a general audience.
Many readers have by now heard of CRISPR – a powerful method of altering or silencing genes that brings down the cost and complexity so that almost any genetics lab can use this technique. CRISPR is actually just the latest of several powerful gene-altering techniques, such as TALEN. CRISPR is essentially a way to target a specific sequence of the DNA, and then deliver a package which does something, like splice the DNA. But you also need to target the correct cells. In a petri dish, this is simple. But in living organism, this is a huge challenge. We have developed several viral vectors that can be targeted to specific cell types in order to deliver the CRIPR (or TALEN), which then targets the specific DNA.
Now I would like to present a different technique I have not previously written about here – alternative splicing. A recent study presents what seems like a significant advance in this technology, so it’s a good time to review it. “Alternative splicing” refers to a natural phenomenon of genetics. Genes are composed of introns and exons. I always thought the nomenclature was counterintuitive, but the exons are actually the part of the gene that gets expressed into a protein. The introns are the part that is not expressed, so they are cut out of the gene when it is being converted into mRNA, and the exons are stitched together to form the sequence that is translated into a protein. Alternative splicing refers to the fact that the way in which the introns are removed and the exons stitched together can vary, creating alternative forms of the resulting protein.
Circa 1999 this can lead to genetic editing that allows people to handle even a nuclear fallout level of radiation and even allow them to handle outer space better.
Rockville, MD — No, it’s not the cockroach, but rather a strain of pink bacteria that can survive 1.5 million rads of gamma irradiation — a dose 3,000 times the amount that would kill a human. This dose of radiation shreds the bacteria’s genome into hundreds of pieces. The organism’s remarkable ability to repair this DNA damage completely in a day and go on living offers researchers tantalizing clues to better understanding the mechanism of cellular repair. Advances in this area could in turn improve our understanding of cancer which is frequently caused by unrepaired DNA damage. Genetically engineering the microbe could lead to improved ways to cleanup pollution and to new industrial processes.
U.S. Department of Energy-funded researchers at The Institute for Genomic Research (TIGR) describe the complete genetic sequence of the bacteria Deinococcus radiodurans in the November 19 issue of Science.
“This is a significant accomplishment,” Secretary of Energy Bill Richardson said. “The Department of Energy began microbial genome work to support bold science and to help meet our unique environment and energy mission needs. Besides the insights into the way cells work, this new research may help provide a new safe and inexpensive tool for some of the nation’s most difficult cleanup challenges.”
Researchers from North Carolina State University have developed a new method for determining which genes are relevant to the aging process. The work was done in an animal species widely used as a model for genetic and biological research, but the finding has broader applications for research into the genetics of aging.
“There are a lot of genes out there that we still don’t know what they do, particularly in regard to aging,” says Adriana San Miguel, corresponding author of a paper on the work and an assistant professor of chemical and biomolecular engineering at NC State.
That’s because this field faces a very specific technical challenge: by the time you know whether an organism is going to live for a long time, it’s old and no longer able to reproduce. But the techniques we use to study genes require us to work with animals that are capable of reproducing, so we can study the role of specific genes in subsequent generations.
On the other, because organisms share the same universal code, they’re vulnerable to outside attacks from viruses and other pathogens—and can transfer their new capabilities to natural organisms, even if it kills them.
Why not build a genetic firewall?
A recent study in Science did just that. The team partially reworked the existing genetic code into a “cipher” that normal organisms can’t comprehend. Similarly, the engineered bacteria lost its ability to read the natural genetic code. The tweaks formed a powerful language barrier between the engineered bacteria and natural organisms, isolating each from sharing genetic information with the other.
Matthew Cobb is a zoologist and author whose background is in insect genetics and the history of science. Over the past decade or so, as CRISPR was discovered and applied to genetic remodeling, he started to get concerned—afraid, actually—about three potential applications of the technology. He’s in good company: Jennifer Doudna, who won the Nobel Prize in Chemistry in 2020 for discovering and harnessing CRISPR, is afraid of the same things. So he decided to delve into these topics, and As Gods: A Moral History of the Genetic Age is the result.
Summing up fears
The first of his worries is the notion of introducing heritable mutations into the human genome. He Jianqui did this to three human female embryos in China in 2018, so the three girls with the engineered mutations that they will pass on to their kids (if they’re allowed to have any) are about four now. Their identities are classified for their protection, but presumably their health is being monitored, and the poor girls have probably already been poked and prodded incessantly by every type of medical specialist there is.
Biohacking is a relatively new practice that focuses on studying and learning about living beings and looking for ways to improve their function.
10 years ago we saw a breakthrough in modern biology.
An American scientist discovered that manipulation of the Cas9 protein resulted in a gene technology worthy of a sci-fi film: CRISPR.
Think of it as a pair of molecular scissors capable of cutting and editing the DNA of humans, animals, plants, bacteria and viruses.