Summary: Researchers have developed a new family of nano-scale capsules capable of carrying CRISPR gene editing tools to different organs of the body before harmlessly dissolving. The capsules were able to enter the brains of mice and successfully edit a gene associated with Alzheimer’s disease.
Source: University of Wisconsin-Madison
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.
Injecting treatments directly into the brain is one way to get around the blood-brain barrier, but it’s an invasive procedure that provides access only to nearby brain tissue.
“The promise of brain gene therapy and genome-editing therapy relies on the safe and efficient delivery of nucleic acids and genome editors to the whole brain,” Gong says.
In a study recently published in the journal Advanced Materials, Gong and her lab members, including postdoctoral researcher and first author of the study Yuyuan Wang, describe a new family of nano-scale capsules made of silica that can carry genome-editing tools into many organs around the body and then harmlessly dissolve.
By modifying the surfaces of the silica nanocapsules with glucose and an amino acid fragment derived from the rabies virus, the researchers found the nanocapsules could efficiently pass through the blood-brain barrier to achieve brain-wide gene editing in mice.
In their study, the researchers demonstrated the capability of the silica nanocapsule’s CRISPR cargo to successfully edit genes in the brains of mice, such as one related to Alzheimer’s disease called amyloid precursor protein gene.
Because the nanocapsules can be administered repeatedly and intravenously, they can achieve higher therapeutic efficacy without risking more localized and invasive methods.
The researchers plan to further optimize the silica nanocapsules’ brain-targeting capabilities and evaluate their usefulness for the treatment of various brain disorders. This unique technology is also being investigated for the delivery of biologics to the eyes, liver and lungs, which can lead to new gene therapies for other types of disorders.

News
Ancient DNA sheds light on evolution of relapsing fever bacteria
Researchers at the Francis Crick Institute and UCL have analyzed ancient DNA from Borrelia recurrentis, a type of bacteria that causes relapsing fever, pinpointing when it evolved to spread through lice rather than ticks, and [...]
Cold Sore Virus Linked to Alzheimer’s, Antivirals May Lower Risk
Summary: A large study suggests that symptomatic infection with herpes simplex virus 1 (HSV-1)—best known for causing cold sores—may significantly raise the risk of developing Alzheimer’s disease. Researchers found that people with HSV-1 were 80% [...]
Nanoparticle-Based Combination Therapy for Resistant Melanoma
A recent study published in Small addresses the persistent difficulty of treating refractory melanoma, an aggressive form of skin cancer that often does not respond to existing therapies. Although diagnostic tools and immunotherapies have improved in [...]
Our DNA May Evolve Much Faster Than Previously Thought
Rapidly mutating DNA regions were mapped using a multi-generational family and advanced sequencing tools. Understanding how human DNA changes over generations is crucial for estimating genetic disease risks and tracing our evolutionary history. However, some of [...]
AI therapy may help with mental health, but innovation should never outpace ethics
Mental health services around the world are stretched thinner than ever. Long wait times, barriers to accessing care and rising rates of depression and anxiety have made it harder for people to get timely help. As a result, governments and health care providers are [...]
Global life expectancy plunges as WHO warns of deepening health crisis Post-COVID
The World Health Organization (WHO) has sounded the alarm on the long-term health repercussions of the COVID-19 pandemic in its newly released World Health Statistics Report 2025. The report reveals a staggering decline in global [...]
Researchers map brain networks involved in word retrieval
How are we able to recall a word we want to say? This basic ability, called word retrieval, is often compromised in patients with brain damage. Interestingly, many patients who can name words they [...]
Melting Ice Is Changing the Color of the Ocean – Scientists Are Alarmed
Melting sea ice changes not only how much light enters the ocean, but also its color, disrupting marine photosynthesis and altering Arctic ecosystems in subtle but profound ways. As global warming causes sea ice in the [...]
Your Washing Machine Might Be Helping Antibiotic-Resistant Bacteria Spread
A new study reveals that biofilms in washing machines may contain potential pathogens and antibiotic resistance genes, posing possible risks for laundering healthcare workers’ uniforms at home. Washing healthcare uniforms at home could be [...]
Scientists Discover Hidden Cause of Alzheimer’s Hiding in Plain Sight
Researchers found the PHGDH gene directly causes Alzheimer’s and discovered a drug-like molecule, NCT-503, that may help treat the disease early by targeting the gene’s hidden function. A recent study has revealed that a gene previously [...]
How Brain Cells Talk: Inside the Complex Language of the Human Mind
Introduction The human brain contains nearly 86 billion neurons, constantly exchanging messages like an immense social media network, but neurons do not work alone – glial cells, neurotransmitters, receptors, and other molecules form a vast [...]
Oxford study reveals how COVID-19 vaccines prevent severe illness
A landmark study by scientists at the University of Oxford, has unveiled crucial insights into the way that COVID-19 vaccines mitigate severe illness in those who have been vaccinated. Despite the global success of [...]
Annual blood test could detect cancer earlier and save lives
A single blood test, designed to pick up chemical signals indicative of the presence of many different types of cancer, could potentially thwart progression to advanced disease while the malignancy is still at an early [...]
How the FDA opens the door to risky chemicals in America’s food supply
Lining the shelves of American supermarkets are food products with chemicals linked to health concerns. To a great extent, the FDA allows food companies to determine for themselves whether their ingredients and additives are [...]
Superbug crisis could get worse, killing nearly 40 million people by 2050
The number of lives lost around the world due to infections that are resistant to the medications intended to treat them could increase nearly 70% by 2050, a new study projects, further showing the [...]
How Can Nanomaterials Be Programmed for Different Applications?
Nanomaterials are no longer just small—they are becoming smart. Across fields like medicine, electronics, energy, and materials science, researchers are now programming nanomaterials to behave in intentional, responsive ways. These advanced materials are designed [...]