How Designer DNA Is Changing Medicine

Normal and sickle-shaped red blood cells.

A genomic revolution is poised to cure sickle cell and other genetic diseases

For as long as he could remember, Razel Colón had known pain. It ripped down his neck and back, shot through his legs and traveled on to his feet, often leaving him writhing and incapacitated. He suffered occasional attacks of “acute chest,” in which breathing suddenly becomes difficult. “It felt like an elephant was sitting on my chest, with tight, tight pain,” Colón tells me. Trips to the emergency department and the hospital were commonplace. “If I was lucky,” he says, “I could stay away for a month.”

Colón, from Hoboken, N.J., is just 19, but the sickle cell disease that produced these effects had been a constant, if unwelcome, companion. But he tells his story now from the perspective of one who has gone a year and a half without that pain. He can do things that previously were out of the question: play basketball, lift weights, swim in cold water. His treatment, says his long-time physician Stacey Rifkin-Zenenberg, a pediatric hematologist-oncologist at Hackensack University Medical Center, “changed him from having the disease to being a carrier.”

Colón’s case represents a point on the curve of an emerging technology that may forever alter our approach to treating diseases like sickle cell. That world, the cutting-edge world of innovative genomic therapies, is once again in the midst of explosive change—and designer DNA lies at the heart of the conversation.

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Hollow nano-objects made of DNA could trap viruses and render them harmless

Lined on the inside with virus-binding molecules, nano-shells made of DNA material bind viruses tightly and thus render them harmless.

by Technical University Munich

To date, there are no effective antidotes against most viral infections. An interdisciplinary research team at the Technical University of Munich (TUM) has now developed a new approach: they engulf and neutralize viruses with nano-capsules tailored from genetic material using the DNA origami method. The strategy has already been tested against hepatitis and adeno-associated viruses in cell cultures. It may also prove successful against corona viruses.

There are antibiotics against dangerous bacteria, but few antidotes to treat acute viral infections. Some infections can be prevented by vaccination, but developing new vaccines is a long and laborious process.

Now an interdisciplinary research team from the Technical University of Munich, the Helmholtz Zentrum München and the Brandeis University (USA) is proposing a novel strategy for the treatment of acute viral infections: The team has developed nanostructures made of DNA, the substance that makes up our genetic material, that can trap viruses and render them harmless.

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Gene editing ‘blocks virus transmission’ in human cells

Researchers in Australia said the tool was effective against viral transmissions in lab tests, adding that they hope soon to begin animal trials on the method

Scientists have used CRISPR gene-editing technology to successfully block the transmission of the SARS-CoV-2 virus in infected human cells, according to research released Tuesday that could pave the way for Covid-19 treatments.

Writing in the journal Nature Communications, researchers in Australia said the tool was effective against viral transmissions in lab tests, adding that they hoped to begin animal trials soon.

CRISPR, which allows scientists to alter DNA sequences and modify gene function, has already shown promise in eliminating the genetic coding that drives the development of children’s cancer.

The team in Tuesday’s study used an enzyme, CRISPR-Cas13b, that binds to relevant RNA sequences on the novel coronavirus and degrades the genome it needs to replicate inside human cells.

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DNAzymes could outperform protein enzymes for genetic engineering

Chemistry professor Yi Lu led a team that developed a technique that allows DNAzymes to cut double-stranded DNA, enabling a wide range of genetic engineering applications.

by Liz Ahlberg Touchstone , University of Illinois at Urbana-Champaign

Move over, gene-editing proteins—there’s a smaller, cheaper, more specific genetic engineering tool on the block: DNAzymes—small DNA molecules that can function like protein enzymes.

Researchers at the University of Illinois Urbana-Champaign have developed a technique that, for the first time, allows DNAzymes to target and cut double-stranded DNA, overcoming a significant limitation of the technology. DNAzymes have been used in biosensing, DNA computing and many other applications. However, when it comes to genetic engineering applications such as gene editing or gene therapy, they have faced a challenge: DNAzymes have only been able to target sites on single-stranded DNA, while the DNA coding for genes in cells is double-stranded. The researchers published their new technique in the Journal of the American Chemical Society.

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New discovery shows human cells can write RNA sequences into DNA

by Thomas Jefferson University

Cells contain machinery that duplicates DNA into a new set that goes into a newly formed cell. That same class of machines, called polymerases, also build RNA messages, which are like notes copied from the central DNA repository of recipes, so they can be read more efficiently into proteins. But polymerases were thought to only work in one direction DNA into DNA or RNA. This prevents RNA messages from being rewritten back into the master recipe book of genomic DNA. Now, Thomas Jefferson University researchers provide the first evidence that RNA segments can be written back into DNA, which potentially challenges the central dogma in biology and could have wide implications affecting many fields of biology.

“This work opens the door to many other studies that will help us understand the significance of having a mechanism for converting RNA messages into DNA in our own cells,” says Richard Pomerantz, Ph.D., associate professor of biochemistry and molecular biology at Thomas Jefferson University. “The reality that a human polymerase can do this with high efficiency, raises many questions.” For example, this finding suggests that RNA messages can be used as templates for repairing or re-writing genomic DNA.

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CRISPR Study Is First to Change DNA in Participants


Written by Meagan Drillinger 

  • For the first time, scientists are altering DNA in a living human.
  • With more research this study could help lead to the development of procedures that can help to correct other genetic disorders.
  • The study uses CRISPR technology, which can alter DNA. 

Researchers from the OHSU Casey Eye Institute in Portland, Oregon, have broken new ground in science, medicine, and surgery — the first gene editing procedure in a living person.

For the first time, scientists are altering DNA in a living human. With more research the study could lead to the development of procedures that can help to correct other genetic disorders.

Known as the BRILLIANCE clinical trial, the procedure is designed to repair mutations in a particular gene that causes Leber congenital amaurosis type 10, also known as retinal dystrophy. It is a genetic condition that results in vision deterioration and has previously been untreatable.

“The Casey Eye Institute performed the first gene editing surgical procedure in a human being in an attempt to prevent blindness from a known genetic mutation,” said Dr. Mark Fromer, ophthalmologist at Lenox Hill Hospital in New York. “The abnormal DNA is removed from a cell with the generating mutation. This will potentially offer sight to people with a form of previously untreatable blindness.”

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A New CRISPR Tool Flips Genes On and Off Like a Light Switch

By Shelly Fan 

CRISPR is revolutionary. It’s also a total brute.

The classic version of the gene editing wunderkind literally slices a gene to bits just to turn it off. It’s effective, yes. But it’s like putting an electrical wire through a paper shredder to turn off a misbehaving light bulb. Once the wires are cut, there’s no going back.

Why not add a light switch instead?

This month, a team from the University of California, San Francisco (UCSF) reimagined CRISPR to do just that. Rather than directly acting on genes—irrevocably dicing away or swapping genetic letters—the new CRISPR variant targets the biological machinery that naturally turns genes on or off.

Translation? CRISPR can now “flip a light switch” to control genes—without ever touching them directly. It gets better. The new tool, CRISPRoff, can cause a gene to stay silent for hundreds of generations, even when its host cells morph from stem cells into more mature cells, such as neurons. Once the “sleeping beauty” genes are ready to wake up, a complementary tool, CRISPRon, flips the light switch back on.

This new technology “changes the game so now you’re basically writing a change [into genes] that is passed down,” said author Dr. Luke Gilbert. “In some ways we can learn to create a version 2.0 of CRISPR-Cas9 that is safer and just as effective.”

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Experimental CRISPR Treatment Cuts Cholesterol in Mice by Up to 57% in a Single Shot

By PETER DOCKRILL

Scientists have improved upon a form of gene-editing therapy, creating an experimental treatment that looks to hold great promise for treating high cholesterol – a diagnosis affecting tens of millions of Americans, and linked to a number serious health complications.

In new research conducted with mice, researchers used an injection of a newly-formulated lipid nanoparticle to deliver CRISPR-Cas9 genome editing components to living animals, with a single shot of the treatment reducing levels of low-density lipoprotein (LDL) cholesterol by up to 56.8 percent.

In contrast, an existing FDA-approved lipid nanoparticle (or LNP; a tiny, biodegradable fat capsule) delivery system could only manage to reduce LDLs by 15.7 percent in testing.

Of course, these results have so far only been demonstrated in mice, so the new therapy will take a lot of further testing before we know it’s both safe and equally effective in humans. But based on these results so far, signs are promising.

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CRISPR Offers the Potential to Live Forever, But to What End?

By Matthew Bacher

Due to the unique consequences of the pandemic, we are able to catch a glimpse of a potential future. One where we sit, plugged into our computers, devoid of physical human connection. What will society look like after the pandemic? Will we continue to stay isolated? Surely advancements in technology have played key roles in prolonging our lives, allowing us to continue to “work” and “socialise,” but to what end? With these newly emerging technologies are we destined to live forever, in a suspended state, in front of the glow of our 4k computer screens? Will gene editing technologies be used to keep us alive forever so that we can binge watch infinite Netflix shows, send meaningless emails and scroll through social media feeds?

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New Research Could Enable Direct Data Transfer From Computers to Living Cells

New-research-could-enable-direct-data-transfers-computers-living-cells

As the modern world produces ever more data, researchers are scrambling to find new ways to store it all. DNAholds promise as an extremely compact and stable storage medium, and now a new approach could let us write digital data directly into the genomes of living cells.

Efforts to repurpose nature’s built-in memory technology aren’t new, but in the last decade the approach has gained renewed interest and seen some major progress. That’s been driven by an explosion of data that shows no signs of slowing down. By 2025, it’s estimated that 463 exabytes will be created each day globally.

Storing all this data could quickly become impractical using conventional silicon technology, but DNA could hold the answer. For a start, its information density is millions of times better than conventional hard drives, with a single gram of DNA able to store up to 215 million gigabytes.

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Researchers discover new way to deliver DNA-based therapies for diseases

researchers-discover-way-to-deliver-DNA-based-therapies

University of Minnesota Twin Cities researchers in the Department of Chemistry have created a new polymer to deliver DNA and RNA-based therapies for diseases. For the first time in the industry, the researchers were able to see exactly how polymers interact with human cells when delivering medicines into the body. This discovery opens the door for more widespread use of polymers in applications like gene therapy and vaccine development.

The research is published in the Proceedings of the National Academy of Sciences (PNAS), a peer-reviewed multidisciplinary scientific journal.

Gene therapy involves altering the genes inside the body’s cells to treat or cure diseases. It requires a carrier that “packages” the DNA to deliver it into the cell—oftentimes, a virus is used as a carrier. Packaging of nucleic acids is also used in vaccines, such as the recently developed messenger RNA (mRNA) COVID-19 vaccine, which is enclosed in a lipid.

The research team is led by chemistry professor Theresa Reineke and associate professor Renee Frontiera. Reineke’s lab synthesizes polymers, which are long-chain molecules that make up plastics, to use for packaging the nucleic acids instead.

“It’s kind of like ordering something from Amazon, and it’s shipped in a box,” Reineke explained. “Things get broken if they’re not delivered in a package. That’s basically what we’re doing here but on a nano-level. We’re taking these really sensitive RNA and DNA cargo that are susceptible to enzymatic degradation, that won’t get to their target unless you have something to protect them.”

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The world’s first DNA ‘tricorder’ in your pocket

by Cold Spring Harbor Laboratory

world's-first-DNA-tricorder-1
Aspyn Palatnick holding the world’s first mobile genetics laboratory at Cold Spring Harbor Laboratory’s 125th anniversary Open House. The combination of the new iPhone app, iGenomics, a DNA analyzer, and Oxford Nanopore’s USB-sized MinION, a DNA sequencer, make genome analysis portable and accessible. Credit: CSHL

Cold Spring Harbor Laboratory (CSHL) scientists developed the world’s first mobile genome sequence analyzer, a new iPhone app called iGenomics. By pairing an iPhone with a handheld DNA sequencer, users can create a mobile genetics laboratory, reminiscent of the “tricorder” featured in Star Trek. The iGenomics app runs entirely on the iOS device, reducing the need for laptops or large equipment in the field, which is useful for pandemic and ecology workers. Aspyn Palatnick programmed iGenomics in CSHL Adjunct Associate Professor Michael Schatz’s laboratory, over a period of eight years, starting when he was a 14-year-old high school intern.

The iPhone app was developed to complement the tiny DNA sequencing devices being made by Oxford Nanopore. Palatnick, now a software engineer at Facebook, was already experienced at building iPhone apps when joining the Schatz laboratory. He and Schatz realized that:

“As the sequencers continued to get even smaller, there were no technologies available to let you study that DNA on a mobile device. Most of the studying of DNA: aligning, analyzing, is done on large server clusters or high-end laptops.”

Schatz recognized that scientists studying pandemics were “flying in suitcases full of Nanopores and laptops and other servers to do that analysis in the remote fields.” iGenomics helps by making genome studies more portable, accessible, and affordable.

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