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Category: bioengineering – Page 134

In October 2019, Liu and his colleagues published a paper in Nature, describing an even newer technology, called prime editing. Prime editing can not only make all twelve of the possible base substitutions, it can also make multiple-base insertions or deletions, without requiring a double-strand break. It achieves this with a multi-step operation that first cuts one strand, then performs the appropriate substitution, insertion, or deletion, and then nicks the second strand to allow the bases on the second strand to be replaced by bases that complement the ones substituted, inserted into or deleted from the first strand. The result is a modified stretch of DNA that had never been completely separated. This has the effect of massively reducing the number of off-target modifications.

This new prime editing variant of CRISPR technology, can make the same corrections to the defects that cause sickle cell disease and beta-thalassemia that standard CRISPR/Cas9 has now made in human subjects, but with less opportunity for unwanted off-target changes. Furthermore, its possible applicability is much wider. The ClinVar database lists over 75,000 pathogenic mutations in the human genome. Of these, over 89% are potentially correctable by prime editing.

From zinc fingers to TALE, to CRISPR/Cas9 to base editing and now to prime editing, progress in gene editing has been accelerating. The next advances are currently being aggressively pursued in laboratories all over the world. It will probably be several years before the therapies that are currently being researched are applied routinely in a clinical setting. However, for people who up until recently have had no hope for a cure to a disease suffered by their child, or even themselves, these are exciting times. The prospect of effective treatments, or even cures, is now a valid cause for hope.

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Bill-gates-thinks-gene-editing-artificial-intelligence-save-world.

Microsoft co-founder Bill Gates has been working to improve the state of global health through his nonprofit foundation for 20 years, and today he told the nation’s premier scientific gathering that advances in artificial intelligence and gene editing could accelerate those improvements exponentially in the years ahead.

“We have an opportunity with the advance of tools like artificial intelligence and gene-based editing technologies to build this new generation of health solutions so that they are available to everyone on the planet. And I’m very excited about this,” Gates said in Seattle during a keynote address at the annual meeting of the American Association for the Advancement of Science.

Such tools promise to have a dramatic impact on several of the biggest challenges on the agenda for the Bill & Melinda Gates Foundation, created by the tech guru and his wife in 2000.

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Dr. Theodore Ho talks about the rapidly expanding possibilities of stem cells to be used in reversing or slowing the aging process. He discusses his previous and current work with the brain, including such methods as tissue clearing, multifiber photometry and optogenetics, and single resolution calcium imaging and control. Dr. Ho is a neuroscientist and stem cell biologist studying the mechanisms and causes of biological aging and potential strategies to slow or reverse them, in order to prevent the onset of age

Associated diseases to help us live healthier and longer lives.

He completed a four-year joint bachelor’s/master’s degree program in.

Human developmental and regenerative biology/bioengineering at.

Harvard University, and he received his PhD in Biophysics from the.

University of California San Francisco, studying stem cell aging in the lab of Dr. Emmanuelle Passegue. In college he developed a nanoparticle drug delivery system, in graduate school he discovered previously unknown mechanisms of cellular and molecular aging of stem cells, and now in the Deisseroth lab he is using optical recording and perturbation of neuronal activity to study neural circuit dynamics that control behavior. This talk was given at a TEDx event using the TED conference format but independently organized by a local community.

Continue reading “Aging and Stem Cells | Theodore Ho | TEDxMiddlebury” | >

The generation of a chemical system capable of replication and evolution is a key objective of synthetic biology. This could be achieved by in vitro reconstitution of a minimal self-sustaining central dogma consisting of DNA replication, transcription and translation. Here, we present an in vitro translation system, which enables self-encoded replication and expression of large DNA genomes under well-defined, cell-free conditions. In particular, we demonstrate self-replication of a multipartite genome of more than 116 kb encompassing the full set of Escherichia coli translation factors, all three ribosomal RNAs, an energy regeneration system, as well as RNA and DNA polymerases. Parallel to DNA replication, our system enables synthesis of at least 30 encoded translation factors, half of which are expressed in amounts equal to or greater than their respective input levels. Our optimized cell-free expression platform could provide a chassis for the generation of a partially self-replicating in vitro translation system.

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Researchers at Columbia Engineering have engineered probiotics to safely deliver immunotherapies within tumors. These include nanobodies against two proven therapeutic targets—PD-L1 and CTLA-4. The drugs are continuously released by bacteria and continue to attack the tumor after just one dose, facilitating an immune response that ultimately results in tumor regression. The versatile probiotic platform can also be used to deliver multiple immunotherapies simultaneously, enabling the release of effective therapeutic combinations within the tumor for more difficult-to-treat cancers like colorectal cancer. The study is published today in Science Translational Medicine.

Antibodies that target immune checkpoints, PD-L1 and CTLA-4, have revolutionized immunotherapy treatments, achieving success in a subset of cancers. However, systemic delivery of these antibodies can also cause substantial side effects with high percentages of patients reporting adverse reactions. Furthermore, although combinations of these therapies are more effective than single therapy regimens, they also produce severe toxicities, sometimes leading to drug discontinuation. The team, led by Tal Danino, assistant professor of biomedical engineering, aimed to address these challenges.

“We wanted to engineer a safe probiotic vehicle capable of delivering immune checkpoint therapies locally to minimize side effects,” says Danino, who is also a member of the Herbert Irving Comprehensive Cancer Center and Data Science Institute. “We also wanted to broaden the versatility of the system by producing a range of immunotherapeutic combinations, including cytokines that could further elicit antitumor immunity, but are otherwise difficult to systemically deliver because of toxicity concerns.”

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Imagine then, the emancipatory potential of genome editing for these millions.

Realizing this potential, however, will require that genome editing meet with societal approval. The typical response right now when you talk to someone about genetic engineering or reproductive technology is a reference to ‘designer babies,’ eugenics, Nazism, and other evils. These arguments have a very powerful emotional hold over many people, but in my opinion, they simply don’t stand up to scrutiny.

Numerous traits, both physical and mental, are too complex to ever be able to engineer, and a Gattaca-like future of ‘designer babies’ is probably just as improbable as time-travel. No serious scientist or ethicist is advocating for government mandated ‘genetic correction’ of the sort Nazism or eugenics implies. As for physical appearance, everyone has their own ideas about the ‘physical ideal.’ Not every visitor to a cosmetic surgeon comes out looking Northern European.

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The diamondback moth is a huge pest. It eats a variety of crops, but is largely resistant to insecticides, resulting in upwards of $5 billion in losses every year.

That could soon change, though, as an international team of researchers has created a strain of genetically engineered diamondback moths that could suppress the pest population in a sustainable way — and they just released them into the wild for the first time.

For the study, published Wednesday in the journal Frontiers in Bioengineering and Biotechnology, the researchers engineered the moths so that when the males of the strain mated with wild females, the female offspring would die during the caterpillar life stage.

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But CRISPR editing — at least as a therapeutic technique in people — has turned out to be more difficult than initially thought. Researchers have documented ways that Cas9, one of the enzymes used in CRISPR gene editing, could trigger immune responses, or cause accidental changes to the genome that would be permanent. RNA editing, by contrast, could allow clinicians to make temporary fixes that eliminate mutations in proteins, halt their production or change the way that they work in specific organs and tissues. Because cells quickly degrade unused RNAs, any errors introduced by a therapy would be washed out, rather than staying with a person forever.

Making changes to the molecular messengers that create proteins might offer flexible therapies for cancer, pain or high cholesterol, in addition to genetic disorders.

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