Category: DNA

Normal and sickle-shaped red blood cells.

• By Carolyn Barber 

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.

Continue reading… “How Designer DNA Is Changing Medicine”

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.

Continue reading… “Hollow nano-objects made of DNA could trap viruses and render them harmless”

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.

Continue reading… “Gene editing ‘blocks virus transmission’ in human cells”

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.

Continue reading… “DNAzymes could outperform protein enzymes for genetic engineering”

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.

Continue reading… “New discovery shows human cells can write RNA sequences into DNA”

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.”

Continue reading… “CRISPR Study Is First to Change DNA in Participants”

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.”

Continue reading… “A New CRISPR Tool Flips Genes On and Off Like a Light Switch”

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.

Continue reading… “Experimental CRISPR Treatment Cuts Cholesterol in Mice by Up to 57% in a Single Shot”

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?

Continue reading… “CRISPR Offers the Potential to Live Forever, But to What End?”