Tag Archives: crispr

what’s new with genetic engineering

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The Week has a roundup.

  • Genetically modified mosquitoes are set to be released in Florida this year and Texas next year. They pass along a gene to their offspring that causes them to die as larvae, potentially wiping out a particularly nasty species that can spread all kinds of disease like Zika, West Nile, and Dengue fever. I know lots of people who have had Dengue fever and it’s nasty. It surprises me we don’t have it in the U.S. yet when we have the mosquitoes now that carry it. Our winter must be just a tiny bit too cool, and how long will that last. The article points out that birds and bats eat mosquitoes. That is certainly true. I tend to think some other little critter will just fill the niche left by the mosquitoes, but we’ll see. Hopefully the birds will have something to eat. As for the bats, I’m kind of mad at them right now.
  • Similar technology is being used to target the diamondback moth and army worm, agricultural pests.
  • University of Georgia made the first genetically modified reptile last year.
  • Genetically modified salmon are already in commercial production.
  • Plants are being modified to absorb more carbon dioxide. (This one actually concerns me. If you release plants that can outcompete native plants that a broad range of insects rely on, there goes your entire food chain. This seems much riskier than targeting one type of insect at a time.)

where gene therapy is heading

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The “J.P. Morgan Healthcare Conference” provided some clues on where biotechnology may be going next.

After decades of setbacks, gene therapy—a loosely defined umbrella term for any technique that uses genes to treat or prevent disease—is finally here. In December, the field got its very first FDA approval with Luxturna, which corrects a defective gene in a rare, inherited retinal disease. With a half dozen more treatments in late-stage trials and an unusually open-minded FDA commissioner in Washington, the industry is expecting a flurry of new approvals this year…

Lambert’s lobbying roadmap for 2018 includes helping insurance companies understand what to do with a new gene therapy like Luxturna, which cures blindness with a single, $850,000 injection into the eye. Ranked by sticker price, it’s the most expensive medicine in America. Spark Therapeutics, the company that makes Luxturna, argues that the six-figure price tag isn’t actually that unreasonable, if you factor in all the costs that patients with the inherited retinal disease would have racked up in a lifetime of seeking better care.

But because their clinical trial patients haven’t been followed long enough to determine if the treatment benefits are actually durable for a whole lifetime, Spark has received significant pushback from insurers. As a result, the company is already exploring a some creative new pricing models. It announced last week that it’s offering a rebate program based on the treatment’s effectiveness at 30 to 90 days and again at 30 months with one East Coast provider, and is in talks about expanding it to other insurers, Spark CEO Jeffrey Marrazzo said at JPM. He said Spark is also in discussions with the Centers for Medicare and Medicaid Services on a multi-year installment plan option. Either of these could soon serve as a model for how gene therapies might be made available to patients without cutting the legs out from under the healthcare system.

The article also mentions one study that has some potentially bad news about the effectiveness of CRISPR in humans, but it sounds like the jury is still out on that.

Change Agent

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From Amazon:

On a crowded train platform, Interpol agent Kenneth Durand feels the sting of a needle—and his transformation begins…

In 2045 Kenneth Durand leads Interpol’s most effective team against genetic crime, hunting down black market labs that perform “vanity edits” on human embryos for a price. These illegal procedures augment embryos in ways that are rapidly accelerating human evolution—preying on human-trafficking victims to experiment and advance their technology.

With the worlds of genetic crime and human trafficking converging, Durand and his fellow Interpol agents discover that one figure looms behind it all: Marcus Demang Wyckes, leader of a powerful and sophisticated cartel known as the Huli jing.

But the Huli jing have identified Durand, too. After being forcibly dosed with a radical new change agent, Durand wakes from a coma weeks later to find he’s been genetically transformed into someone else—his most wanted suspect: Wyckes.

Now a fugitive, pursued through the genetic underworld by his former colleagues and the police, Durand is determined to restore his original DNA by locating the source of the mysterious—and highly valuable—change agent. But Durand hasn’t anticipated just how difficult locating his enemy will be. With the technology to genetically edit the living, Wyckes and his Huli jing could be anyone and everyone—and they have plans to undermine identity itself.

using CRISPR to create new crops

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This article in Trends in Plant Science (which I know you’ve seen, since it flies off the news stands) argues that CRISPR should be used to create entirely new crops out of wild plants, mimicking the process that created our most common cereal crops over thousands of years.

Of the more than 300 000 plant species that exist, less than 200 are commercially important, and three species – rice, wheat, and maize – account for the major part of the plant-derived nutrients that humans consume.

Plants with desirable traits, such as perennials with extensive root systems and nitrogen-fixing plants, are currently being domesticated as new crops…

Several traits in crops that were crucial for their domestication are caused by mutations that can be reproduced by genome-editing techniques such as CRISPR/Cas9, offering the potential for accelerated domestication of new crops.

containing technologies

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This post is about CRISPR and gene drive, which are interesting in their own right. What I am going to quote is the author’s ideas on how to develop a promising but potentially dangerous technology responsibly:

For starters, public notification and broadly inclusive discussions should always precede and inform development of gene drive interventions in the lab. A clear description of the potential impact of an experiment – as my colleagues and I have provided for the technology as a whole – must be followed by transparency throughout the development process. This community-guided approach to research provides opportunities to identify and address potential problems and concerns during development. If a perceived problem cannot be adequately addressed, researchers should be prepared to terminate the project…

Another feature of a responsible approach would be a commitment by scientists to evaluate each proposed gene drive intervention – say, immunizing mice so that they cannot transmit Lyme disease to ticks – individually, rather than making a blanket decision on the technology as a whole. After all, the benefits and risks of each intervention would be entirely different.

A final safeguard against the irresponsible development of gene drive technology is to ensure that early interventions are developed exclusively by governments and nonprofit organizations. Given the potential of financial incentives to skew the design and results of safety tests, keeping the profit motive out of the development and decision-making processes will encourage balanced assessments.

CRISPR

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Here’s some more info on CRISPR, a genetic engineering technique some people are saying will be revolutionary.

 The Bacterial Origins of the CRISPR Genome-Editing Revolution
Sontheimer Erik J. and Barrangou Rodolphe. Human Gene Therapy. July 2015, 26(7): 413-424. doi:10.1089/hum.2015.091.

Like most of the tools that enable modern life science research, the recent genome-editing revolution has its biological roots in the world of bacteria and archaea. Clustered, regularly interspaced, short palindromic repeats (CRISPR) loci are found in the genomes of many bacteria and most archaea, and underlie an adaptive immune system that protects the host cell against invasive nucleic acids such as viral genomes. In recent years, engineered versions of these systems have enabled efficient DNA targeting in living cells from dozens of species (including humans and other eukaryotes), and the exploitation of the resulting endogenous DNA repair pathways has provided a route to fast, easy, and affordable genome editing. In only three years after RNA-guided DNA cleavage was first harnessed, the ability to edit genomes via simple, user-defined RNA sequences has already revolutionized nearly all areas of biological science. CRISPR-based technologies are now poised to similarly revolutionize many facets of clinical medicine, and even promise to advance the long-term goal of directly editing genomic sequences of patients with inherited disease. In this review, we describe the biological and mechanistic basis for these remarkable immune systems, and how their engineered derivatives are revolutionizing basic and clinical research.