Category: DNA

DNA, or deoxyribonucleic acid, is the molecular foundation that carries genetic information in living organisms, using its double helix structure to transcribe and amplify this information. Scientists are keen on developing artificial molecular systems that can rival or even surpass the functionality of DNA. Among these systems, double-helical foldamers stand out as promising candidates.

Helical foldamers are synthetic molecules designed to fold into well-defined helical structures, similar to those found in proteins and nucleic acids. These molecules have gained attention for their potential as stimuli-responsive materials, tunable chiral systems, and cooperative supramolecular structures due to their unique chiral and conformational switching properties. Double-helical foldamers, in particular, exhibit enhanced chiral properties and the ability to transmit chiral information from one strand to another, opening the door to applications in replication-like processes found in nucleic acids. However, controlling the chiral switching of these artificial molecules has been challenging, due to the need for a delicate balance between stability and dynamic properties.

Continue reading… “Breakthrough in Artificial DNA: Double-Helical Monometallofoldamers with Controllable Chiral Switching”

According to a recent publication in Nature Biotechnology, researchers from New York University, Columbia Engineering, and the New York Genome Center have discovered that artificial intelligence (AI) can accurately predict the on- and off-target activity of CRISPR tools that target RNA instead of DNA. This groundbreaking study combines a deep learning model with CRISPR screens, enabling researchers to control the expression of human genes in various ways. This precise gene control could lead to the development of novel CRISPR-based therapies.

CRISPR technology has garnered significant attention due to its versatility in biomedical applications, ranging from treating genetic diseases like sickle cell anemia to enhancing the characteristics of crops. Traditionally, CRISPR targets DNA using the Cas9 enzyme. However, scientists have recently uncovered an alternative form of CRISPR that targets RNA using the Cas13 enzyme.

Continue reading… “AI Enables Predictive Power for RNA-Targeting CRISPR Tools, Facilitating Precise Gene Control”

• Aging is a natural part of life that changes the body in ways we sometimes might not like. 
• Researchers from Harvard Medical School believe that epigenetic changes — and not just changes to the DNA — affect aging.
• This view is supported by experiments where epigenetic changes caused mice to first age and the reversal of the induced changes caused reverse aging. 

Aging is a life process everyone goes through. As we age, the body changes in different ways — sometimes good and sometimes not as good as we might like. 

Scientists have looked for ways to slow down, stop, or reverse the aging process. While research and medical advances have helped increase life expectancy, aging continues. 

For many years, most researchers have believed changes to a body’s DNA — called mutations — are a leading cause of aging. 

Now a team led by researchers from Harvard Medical School finds support for an alternative hypothesis: it is the changes that affect the expression of the DNA — called epigenetics — that affect aging. Scientists demonstrated this via a mouse model where changes in epigenetic information caused mice to first age and then reverse aging. 

The study appears in the journal Cell. 

Continue reading… “Scientists reversed aging in mice. Is it possible in humans?”

Researchers at UW–Madison have engineered silica nanocapsules to cross the blood-brain barrier in mice to deliver brain-wide gene editing therapy for Alzheimer’s disease. 

By Laura Red Eagle

Gene therapies have the potential to treat neurological disorders like Alzheimer’s and Parkinson’s diseases, but they face a common barrier — the blood-brain barrier. Now, researchers at the University of Wisconsin–Madison have developed a way to move therapies across the brain’s protective membrane to deliver brain-wide therapy with a range of biological medications and treatments.

“There is no cure yet for many devastating brain disorders,” says Shaoqin “Sarah” Gong, UW–Madison professor of ophthalmology and visual sciences and biomedical engineering and researcher at the Wisconsin Institute for Discovery. “Innovative brain-targeted delivery strategies may change that by enabling noninvasive, safe and efficient delivery of CRISPR genome editors that could, in turn, lead to genome-editing therapies for these diseases.”

CRISPR is a molecular toolkit for editing genes (for example, to correct mutations that may cause disease), but the toolkit is only useful if it can get through security to the job site. The blood-brain barrier is a membrane that selectively controls access to the brain, screening out toxins and pathogens that may be present in the bloodstream. Unfortunately, the barrier bars some beneficial treatments, like certain vaccines and gene therapy packages, from reaching their targets because in lumps them in with hostile invaders.

Continue reading… “New nanocapsules deliver therapy brain-wide, edit Alzheimer’s gene in mice”

Predicted 3D structure for a segment of human genomic DNA.

Newly developed artificial intelligence (AI) programs accurately predicted the role of DNA’s regulatory elements and three-dimensional (3D) structure based solely on its raw sequence, according to two recent studies in Nature Genetics. These tools could eventually shed new light on how genetic mutations lead to disease and could lead to new understanding of how genetic sequence influences the spatial organization and function of chromosomal DNA in the nucleus, said study author Jian Zhou, Ph.D., Assistant Professor in the Lyda Hill Department of Bioinformatics at UTSW.

“Taken together, these two programs provide a more complete picture of how changes in DNA sequence, even in noncoding regions, can have dramatic effects on its spatial organization and function,” said Dr. Zhou, a member of the Harold C. Simmons Comprehensive Cancer Center, a Lupe Murchison Foundation Scholar in Medical Research, and a Cancer Prevention and Research Institute of Texas (CPRIT) Scholar.

Only about 1% of human DNA encodes instructions for making proteins. Research in recent decades has shown that much of the remaining noncoding genetic material holds regulatory elements – such as promoters, enhancers, silencers, and insulators – that control how the coding DNA is expressed. How sequence controls the functions of most of these regulatory elements is not well understood, Dr. Zhou explained.

Continue reading… “AI Tools Can Predict DNA Structure and Regulation”

By Mert Erdemir

An international team of scientists from the U.K., U.S., and Singapore is working together to develop an injectable cure for genetic heart conditions by rewriting DNA. The team named CureHeart has been awarded a £30 million grant from the British Heart Foundation (BHF).

The researchers will employ precision genetic techniques in the heart for the first time with the aim of silencing defective genes and develop and test the first treatment for genetic heart diseases. Animal tests had already proven before that the techniques work.

“This is a defining moment for cardiovascular medicine,” said Professor Sir Nilesh Samani, the BHF’s medical director. “Not only could CureHeart be the creators of the first cure for inherited heart muscle diseases by tackling killer genes that run through family trees, it could also usher in a new era of precision cardiology.”

Continue reading… “In a world first, scientists rewrite DNA to cure ‘genetic heart conditions’”

Image of patient derived podocyte kidney cells repaired with novel baculovirus-vectored approach pioneered by the Berger team. Podocin (coloured in green) is restored to the cell surface as in healthy podocytes. Credit: Dr Francesco Aulicino.Read time:  4 minutesDownload Article

Genetic mutations which cause a debilitating hereditary kidney disease affecting children and young adults have been fixed in patient-derived kidney cells using a potentially game-changing DNA repair-kit. The advance, developed by University of Bristol scientists, is published in Nucleic Acids Research.

In this new study, the international team describe how they created a DNA repair vehicle to genetically fix faulty podocin, a common genetic cause of inheritable Steroid Resistant Nephrotic Syndrome (SRNS).

Podocin is a protein normally located on the surface of specialised kidney cells and is essential for kidney function. Faulty podocin, however, remains stuck inside the cell and never makes it to the surface, terminally damaging the podocytes. Since the disease cannot be cured with medications, gene therapy which repairs the genetic mutations causing the faulty podocin offers hope for patients.

Continue reading… “DNA Repair Kit Successfully Fixes Hereditary Disease in Cells”

 By Emily Henderson, B.Sc.

An injectable cure for inherited heart muscle conditions that can kill young people in the prime of their lives could be available within a few years, after an international team of researchers were announced as the winners of the British Heart Foundation’s Big Beat Challenge.

The global award, at £30m, is one of the largest non-commercial grants ever given and presents a “once in a generation opportunity” to provide hope for families struck by these killer diseases.

The winning team, CureHeart, will seek to develop the first cures for inherited heart muscle diseases by pioneering revolutionary and ultra-precise gene therapy technologies that could edit or silence the faulty genes that cause these deadly conditions.

The team, made up of world-leading scientists from the UK, US and Singapore, was selected by an International Advisory Panel chaired by Professor Sir Patrick Vallance, Chief Scientific Advisor to the UK Government.

Inherited heart muscle diseases can cause the heart to stop suddenly or cause progressive heart failure in young people. Every week in the UK, 12 people under the age of 35 die of an undiagnosed heart condition1, very often caused by one of these inherited heart muscle diseases, also known as genetic cardiomyopathies. Around half of all heart transplants are needed because of cardiomyopathy and current treatments do not prevent the condition from progressing.

Continue reading… “Ultra-precise gene therapy technologies could edit or silence faulty genes causing fatal heart diseases”