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Category: bioengineering – Page 68

Base Editing Beats Other Genome Editing Strategies for Treating Sickle Cell Disease

The findings suggest that adenosine base editing raised the expression of fetal hemoglobin to higher, more stable, and more uniform levels than other genome editing technologies that use CRISPR/Cas9 nuclease in human hematopoietic stem cells.

“Ultimately, we showed that not all genetic approaches are equal,” said Jonathan Yen, PhD, genome engineering group director at St. Jude Children’s Research Hospital. “Base editors may be able to create more potent and precise edits than other technologies. But we must do more safety testing and optimization.”

SCD and beta-thalassemia are blood disorders caused by mutations in the gene encoding hemoglobin affecting millions of people. Restoring gene expression of an alternative hemoglobin subunit active in a developing fetus has previously shown therapeutic benefit in SCD and beta-thalassemia patients. The researchers wanted to find and optimize genomic technology to edit the fetal hemoglobin gene.

Adult hemoglobin, expressed primarily after birth, contains four protein subunits—two beta-globin and two alpha-globin. Mutations in the beta-globin gene cause sickle cell disease and beta-thalassemia. But humans have another hemoglobin subunit gene (gamma-globin), which is expressed during fetal development instead of beta-globin. Gamma-globin combines with alpha-globin to form fetal hemoglobin. Normally around birth, gamma-globin expression is turned off, and beta-globin is turned on, switching from fetal to adult hemoglobin. Genome editing technologies can introduce mutations that turn the gamma-globin gene back on, thereby increasing fetal hemoglobin production, which can effectively substitute for defective adult hemoglobin production.

Scientists Dramatically Extend Cell Lifespan in Anti-Aging Breakthrough

“A major highlight of the work is our approach to achieve longevity: using computers to simulate the natural aging system and guide the design and rational engineering of the system to extend lifespan,” Hao told Motherboard. “This is the first time this computationally-guided engineering-based approach has been used in aging research. Our model simulations actually predicted that an oscillator can double the lifespan of the cell, but we were happily surprised that it actually did in experiments.”

The study is part of a growing corpus of mind-boggling research that may ultimately stave off some of the unpleasant byproducts of aging until later in life, while boosting life expectancy in humans overall. Though countless hurdles have to be cleared before these treatments become a reality, Hao thinks his team’s approach could eventually be applied to humans.

“I don’t see why it cannot be applied to more complex organisms,” Hao said. “If it is to be introduced to humans, then it will be a certain form of gene therapy. Of course it is still a long way ahead and the major concerns are on ethics and safety.”

Researchers make breakthrough in functional human tissue 3D printing

Nominations are now open for the 3D Printing Industry Awards 2023. Who are the leaders in 3D printing? Find out on November 30th when the winners across twenty categories will be announced during a London-based live awards ceremony.

A team of scientists from the University of Sydney and the Children’s Medical Research Institute (CMRI) at Westmead have leveraged 3D photolithographic printing to fabricate functional human tissues that accurately mimic an organ’s architecture.

The researchers utilized bioengineering and cell culture techniques to instruct stem cells derived from blood cells and skin cells to become specialized. These specialized cells can then form organ-like structures.

Pluripotent stem cell-based therapies and their path to the clinic

Welcome to this special issue, focusing on the potential of pluripotent stem cell (PSC)-based therapies and their paths toward clinical application. Since the establishment of human embryonic stem (ES) and induced pluripotent stem (iPS) cells in 1998 and 2007, respectively, significant progress has been made in differentiating PSCs into a broad range of somatic cells. We are now closer than ever before to having highly functional PSC-derived somatic cells at purity for transplantation therapies to complement damaged or diseased organs and restore their physiologic functions. Like organ transplantation, PSC-based therapies have the potential to regenerate damaged organs that cannot otherwise be healed by using small-molecule or antibody-based drugs.

In this issue, Kobold et al. present an overview of the history and current status of clinical studies utilizing human PSCs. Since the early 2010s, many clinical studies employing human ES cells have been initiated. By 2018, the number of such studies using human iPS cells had skyrocketed. Many PSC-based therapies are currently being tested to treat various pathologic conditions, including different neoplasms and diseases of the eye, adnexa, and circulatory system. However, there are still many diseases that require further efforts to interrogate the true potential of PSC-based therapies. To advance the use of PSC-based therapy to treat a wider range of pathologic conditions in the future, we must continue with extensive basic and clinical research to establish both efficacy and safety for such new therapies.

Although clinical research on PSC-based therapy for liver diseases has not received as much attention, there is much hope for it to become a real alternative to living-donor liver transplantation. Cardinale et al. provided a comprehensive summary of the recent studies on cell-based therapy for liver diseases. In addition, artificial livers generated through bioengineering efforts are now considered to be a viable option. Aside from traditional cell or organ transplantation to restore impaired liver function, transplantation aimed at treating the microenvironment, such as inflammation, in the liver is also an effective therapeutic strategy. Concurrent research efforts in both basic and clinical studies will be crucial in making PSC-based therapy for liver diseases a reality.

Q&A: Growing Steaks in the Lab

Physicist Luke MacQueen combines tissue engineering with stem cell technologies to produce synthetic meat whose texture mimics that of natural meat.

Winston Churchill—the well-known wartime leader and lesser-known Nobel Laureate in Literature—published an essay in 1931 in The Strand Magazine in which he imagined the future “Fifty Years Hence.” Many of his predictions turned out to be prophetic—wireless telephones, television, and nuclear power—while others read like science fiction. But one of his futuristic ideas—growing meat in a lab—may just be a few years away, if Luke MacQueen of Harvard University has his way.

Bioelectricity Gives Biologists a Jolt

We’ve explored bioelectricity in cells. We’ve looked at bioelectricity within the human body. Now, functional use of “electrical engineering” is being found in the realms between.

Physicists learn about electrostatics, the laws governing stationary charges. Then they learn about electrodynamics, the laws governing moving charges. Biologists are finding that life utilizes both systems of laws at all scales, from within the cell to tissues, organs, and entire organisms. Here are some recent discoveries in the emerging science of bioelectricity.

How does that tick jump from its twig onto your clothing as you walk through brush? The answer, says Current Biology, is by hopping on an electrostatic bullet train. A cow or other host animal walking through the bushes carries a net static charge. The tick, regardless of its own charge polarity, is “pulled by these electric fields across air gaps of several body lengths.”

Durham gene editing firm strikes big deal; it’s ‘right-sized;’ 80 employees exit

DURHAM – A big licensing deal potentially worth hundreds of millions of dollars with an Austrlia-based company at the same time also has triggered what Precision Biosciences calls a “right-sized” organization of the company.

“Prior to the announcement, we had 190 employees, with 110 going forward with Precision. Most of the 80 employees went with Imugene, with the remainder parting ways with a reduction in force,” Mei Burris, director of investor relations and finance for the company,” told WRAL TechWire.

What “right-sized” means was not immediately explained in the company’s announcement Tuesday night after the markets closed. The company’s stock is trading at under $1 and it lost $12 million in its most recent quarter ending June 30.

The World’s First Genetically Engineered Wheat Is Here

In October 2020, Argentina approved the world’s first genetically engineered wheat for cultivation and consumption. Production expanded dramatically in 2021, and will continue to expand in 2022, after Argentina received regulatory approval in late 2021 for exports to Brazil, a major consumer of Argentina’s wheat.

The lessons from Argentina’s experience are important as other countries decide whether they want to follow suit. Argentina’s genetically engineered, drought-tolerant wheat — named HB4 — could have large environmental benefits, but other countries’ choices will determine their scale.

Argentina is increasingly struggling with drought and saw an opportunity for HB4 wheat to help stabilize production and revenue. Yields have been steadily decreasing since 2017, partially due to drought, with the 2020/21 season yields the second-lowest in ten years. Yields in the 2021/22 season bounced back thanks to sufficient rainfall at critical times. HB4 wheat, genetically engineered to be drought resistant, can help protect against such variability by maintaining high yields even under drought conditions. HB4’s drought resistance gene comes from sunflowers, so it qualifies as transgenic — containing genes from a different species — and therefore as bioengineered, genetically modified, or a GMO.

Could Rotifers be the Next Great Laboratory Model?

Rotifers are multicellular, microscopic marine animals that live in soils and freshwater environments. They are transparent and can be easily grown in large numbers. As such, they have been used in some laboratories as research subjects for many years. Now scientists have found a way to manipulate the rotifer genome, which can make them far more useful for many different research applications.

In new work reported in PLOS Biology, scientists used the CRISPR-Cas9 gene editing tool to alter two rotifer genes. These edits were then passed down to future generations of rotifers. This effort can now help others use these organisms in their laboratories.

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