Nucleic acid therapies involving DNA or RNA have significant potential to treat genetic disorders, infectious diseases, and cancer; however, research suggests that less than 1% of injected nucleic acid doses reach target cells in an active form. An article in WIREs Nanomedicine and Nanobiotechnology highlights two of the major barriers to this therapeutic strategy, and how they might be overcome.
Delivery to the target cell and transport to the sub-cellular compartment where the nucleic acids are therapeutically active represent the most significant challenges to address. The authors explore how nanoparticle delivery systems can be modified with targeting molecules to increase accumulation in specific cells, and how the composition of the nanoparticle can be engineered to manipulate or disrupt cellular membranes and facilitate delivery to the optimal sub-cellular compartments.
“Controlling the sub-cellular delivery of DNA and RNA is the next major frontier for biotherapeutics. If we can overcome these barriers, DNA and RNA technology has the potential to revolutionize the treatment of a range of diseases,” said corresponding author Angus P.R. Johnston, Ph.D., of Monash University, in Australia.
News
Human brain map contains never-before-seen details of structure
A cubic millimeter of brain tissue may not sound like much. But considering that tiny square contains 57,000 cells, 230 millimeters of blood vessels, and 150 million synapses, all amounting to 1,400 terabytes of [...]
AI Ethics Surpass Human Judgment in New Moral Turing Test
AI’s ability to address moral questions is improving, which prompts further considerations for the future. A recent study revealed that when individuals are given two solutions to a moral dilemma, the majority tend to [...]
AstraZeneca removes its Covid vaccine worldwide after rare side effect
AstraZeneca withdraws COVID-19 vaccine: The pharmaceutical giant AstraZeneca's COVID-19 vaccine is being withdrawn worldwide after the company acknowledged for the first time in court documents that it can cause a rare and dangerous side [...]
AI Transforms Drug Discovery With Faster, Safer Cancer Treatments
The new platform helped UC San Diego scientists synthesize 32 potential multi-target cancer drugs. Scientists at UC San Diego have developed a machine learning algorithm to simulate the time-consuming chemistry involved in the earliest phases of [...]
Nanotech opens door to future of insulin medication
An international team, led by researchers from Australia, have developed a system using nanotechnology that could allow people with diabetes to take oral insulin in the future. The researchers say the new insulin could [...]
COVID-19’s Impact on Child Development More Subtle Than Expected
Researchers at Johns Hopkins Children’s Center express that the study results offer a sense of reassurance, instilling “guarded optimism” regarding the developmental outcomes of young children who have been exposed to restrictions stemming from [...]
New Hope for Neurological Disorders: Scientists Have Discovered How an Essential Nutrient Enters the Brain
Researchers have discovered the process by which dietary choline crosses the blood-brain barrier. This breakthrough has potential applications in enhancing drug delivery to the brain for treating neurological disorders. A researcher from the University of [...]
Developing a tech platform for fast, robust series production of nanoparticles
Nucleic acid-based medications such as mRNA vaccines offer tremendous potential for medicine and are opening up new therapeutic approaches. These active ingredients must be enclosed inside nanoparticles to ensure that they get to where [...]
Unlocking Medical Mysteries: Tyche AI’s Revolutionary Insight Into Ambiguity
By providing plausible label maps for one medical image, the Tyche machine-learning model could help clinicians and researchers capture crucial information. In biomedicine, segmentation involves annotating pixels from an important structure in a medical [...]
Scientists Decode the Molecular Impact of Exercise
MoTrPAC examined the molecular effects of exercise on 2,600 volunteers, incorporating factors like age, race, and gender diversity. Building upon research in rats, MoTrPAC discovered over 35,000 biological molecules responding to endurance exercise and [...]
Prostate Cancer Breakthrough: Urine Test Avoids Unnecessary Biopsies
A study in JAMA Oncology reveals that MyProstateScore 2.0, a new urine test analyzing 18 genes, surpasses PSA in detecting significant prostate cancers and could reduce unnecessary biopsies by up to 42%. A new urine test [...]
Wake Up and Die: New Brain Cell Discovery Could Unlock Alzheimer’s Secrets
This uncommon process is more frequently observed in neurodegenerative diseases and could offer insights into disease mechanisms. According to a new study published in PLOS Biology by Kim Hai-Man Chow and colleagues from the Chinese University [...]
Challenging Old Theories: Innovative Microscopy Exposes New Alzheimer’s Treatment Pathways
Researchers at UC San Diego have utilized advanced imaging techniques to explore the metabolic processes behind Alzheimer’s disease, leading to potential new strategies for treatment. Alzheimer’s disease, the most common type of dementia, significantly impairs [...]
Cambridge Scientists Discover Simple “Twist” That Supercharges Clean Fuel Generation
Scientists have discovered a method to super-charge the ‘engine’ of sustainable fuel generation – by giving the materials a little twist. The researchers, led by the University of Cambridge, are developing low-cost light-harvesting semiconductors that power devices [...]
Completely New Use Discovered – This Traditional Herb Has Remarkable Nerve Regenerative Properties
Blessed thistle (Cnicus benedictus), a member of the Asteraceae family, thrives in our climate. This plant has been utilized for centuries as a medicinal herb, often consumed as an extract or tea to support [...]
Scientists study lipids cell by cell, making new cancer research possible
Imagine being able to look inside a single cancer cell and see how it communicates with its neighbors. Scientists are celebrating a new technique that lets them study the fatty contents of cancer cells, [...]