Gene Therapy News

June 26, 2025

Top Headlines

Iron Overload: The Hidden Culprit Behind Early Alzheimer’s in Down Syndrome

June 21, 2025 — USC researchers have uncovered a hidden driver behind the early and severe onset of Alzheimer's in people with Down syndrome: iron overload in the brain. Their study revealed that individuals with ...

Scientists Design Gene Delivery Systems for Cells in the Brain and Spinal Cord

May 21, 2025 — Research teams have created a versatile set of gene delivery systems that can reach different neural cell types in the human brain and spinal cord with exceptional accuracy. These delivery systems ...

CAR-T Cell Therapy for Cancer Causes 'Brain Fog,' Study Shows

May 12, 2025 — Cancer treatment with a cell-based immunotherapy causes mild cognitive impairment, a Stanford Medicine team found. They also identified compounds that could treat ...

Researchers Map 7,000-Year-Old Genetic Mutation That Protects Against HIV

May 9, 2025 — Modern HIV medicine is based on a common genetic mutation. Now, researchers have traced where and when the mutation arose -- and how it protected our ancestors from ancient ...

Scientists Engineer Precision Tool for Mitochondrial DNA Manipulation

May 4, 2025 — Many mitochondrial diseases have been difficult to study and treat due to the inherent challenges in accessing mitochondrial DNA (mtDNA). Now, researchers have optimized mitochondrial-targeted compounds that can selectively modify the ratio of ...

Structure Dictates Effectiveness, Safety in Nanomedicine

Apr. 25, 2025 — Historically, small molecule drugs have been precisely designed down to the atomic scale. Considering their relatively large complex structures, nanomedicines have lagged behind. Researchers argue this precise control should be applied to optimize ...

Key Enzyme in Lipid Metabolism Linked to Immune System Aging

Apr. 19, 2025 — New research suggests that decreased activity of ELOVL2 -- a key enzyme in lipid metabolism -- accelerates white blood cell aging and alters genes associated with the onset of blood ...

New Type of Handheld Detector for All Types of Ionizing Radiation Improves Radiation Safety

Apr. 15, 2025 — Scientists have developed a new type of handheld multi-purpose radiation detector that comprehensively detects all types of ionizing radiation. The device can be used by industrial and medical ...

Preventing Onset and Development of Heart Failure With Preserved Ejection Fraction

Apr. 15, 2025 — An international team of researchers has discovered a natural mechanism that protects the heart from heart failure with preserved ejection fraction (HFpEF), a serious condition in need of effective ...

Engineering Smart Delivery for Gene Editors

Apr. 9, 2025 — A research team has developed an advanced delivery system that transports gene-editing tools based on the CRISPR/Cas9 gene-editing system into living cells with significantly greater efficiency than before. Their technology, ENVLPE, uses engineered ...

PEPITEM Sequence Shows Effects in Psoriasis, Comparable to Steroid Cream

Apr. 3, 2025 — Scientists have shown that a sequence of just three amino acids may reduce the severity of psoriasis, when applied topically in an emollient ...

How a Critical Enzyme Keeps Potentially Dangerous Genes in Check

Mar. 30, 2025 — The new study reveals a remarkable way that cells keep us safe from transposable elements (TEs) gone wild. The researchers found that cells have taken advantage of an entire protein network to repress TE activity and keep themselves ...

Latest Headlines

updated 1:09pm EDT

New AI Model Reveals Your True Biological Age from 5 Drops of Blood

Mar. 26, 2025 — A research team has developed a new system to estimate a person's biological age -- a measure of how well their body has aged, rather than just counting the years since birth. Using just five drops ...

Researchers Identify a Gene to Guide Novel Therapeutics of Nonalcoholic Fatty Liver Diseases

Mar. 7, 2025 — Investigators have found that the gene Asah1 plays a crucial protective role in preventing the progression of nonalcoholic fatty liver diseases ...

A Protein from Tiny Tardigrades May Help Cancer Patients Tolerate Radiation Therapy

Feb. 26, 2025 — Drawing inspiration from the tardigrade, researchers developed a new strategy that may protect cancer patients from the side effects of radiation ...

DNA Origami Suggests Route to Reusable, Multifunctional Biosensors

Feb. 24, 2025 — A team has used a process known as DNA origami to make electrochemical sensors that can quickly detect and measure ...

Digging Into a Decades-Old Hepatitis B Mystery Suggests a New Potential Treatment

Feb. 20, 2025 — In their effort to answer a decades-old biological question about how the hepatitis B virus (HBV) is able to establish infection of liver cells, ...

Scientists Develop Innovative DNA Hydrogels for Sustained Drug Release

Feb. 18, 2025 — DNA hydrogels are biocompatible drug delivery systems for targeted therapeutic interventions. Conventional DNA hydrogels, formed with many DNA nanostructure units, lead to increased preparation costs ...

New Lipid Nanoparticle Platform Delivers mRNA to the Brain Through the Blood-Brain Barrier

Feb. 18, 2025 — Scientists have developed a lipid nanoparticle system capable of delivering messenger RNA (mRNA) to the brain via intravenous injection, a challenge ...

Epidural Steroid Injections for Chronic Back Pain

Feb. 14, 2025 — Scientists have developed a new systematic review to summarize for neurologists and other clinicians the evidence for epidural steroid injections and whether they reduce pain and disability for ...

Mystery Solved: New Study Reveals How DNA Repair Genes Play a Major Role in Huntington's Disease

Feb. 11, 2025 — A new study has discovered in mouse models that genes associated with repairing mismatched DNA are critical in eliciting damages to neurons that are ...

A Light-Activated Drug to Fight Psoriasis

Feb. 3, 2025 — Psoriasis is a chronic skin disease that manifests itself mainly with skin symptoms (dryness, itching, scaly skin, abnormal patches and plaques). It affects about 2% of the population and is mediated ...

Escaping the Endosome: Bend Lipids Improve LNP mRNA Delivery and Gene Editing

Jan. 28, 2025 — A new class of lipids improve the rates at which lipid nanoparticles (LNPs) successfully deliver RNA therapeutics and gene editing tools, promising to increase their effectiveness of such ...

Researchers Pioneer DNA-Tagged Gold Nanoparticles for Targeted Cancer Treatment

Jan. 28, 2025 — A team of researchers has developed a novel method to enhance the precision of cancer treatment using gold nanoparticles tagged with DNA ...

Textbooks Need to Be Rewritten: RNA, Not DNA, Is the Main Cause of Acute Sunburn

Jan. 17, 2025 — Sunburn has traditionally been attributed to UV-induced DNA damage. However, a new study reveals that RNA, another vital cellular molecule, plays a major role in triggering acute sunburn ...

Florescent Probes Illuminate Cholesterol and Alzheimer's Research

Jan. 17, 2025 — Cutting-edge fluorescent cholesterol probes now enable scientists to visualize cholesterol movement and distribution in live cells with unprecedented detail. By combining computer simulations with ...

Study Finds Surprising Way That Genetic Mutation Causes Huntington's Disease, Transforming Understanding of the Disorder

Jan. 16, 2025 — Scientists have discovered a surprising mechanism by which the inherited genetic mutation known to cause Huntington's disease leads to the death of brain cells. The findings change the ...

DNA Damage Can Last Unrepaired for Years, Changing Our View of Mutations

Jan. 15, 2025 — While most known types of DNA damage are fixed by our cells' in-house DNA repair mechanisms, some forms of DNA damage evade repair and can persist for many years, new research shows. This means ...

DNA Nanorobots Can Alter Artificial Cells

Jan. 13, 2025 — Scientists have succeeded in controlling the structure and function of biological membranes with the help of 'DNA origami'. The system they developed may facilitate the transportation of ...

Shape-Changing Device Helps Visually Impaired People Perform Location Task as Well as Sighted People

Dec. 10, 2024 — A groundbreaking piece of navigation technology that uses the ability to sense information through touch can help people with visual impairment perform a location task as well as sighted people, ...

First New Treatment for Asthma Attacks in 50 Years

Nov. 27, 2024 — A recent trial finds an injection given during some asthma and COPD attacks is more effective than the current treatment of steroid tablets, reducing the need for further treatment by ...

Cellular Traffic Congestion in Chronic Diseases Suggests New Therapeutic Targets

Nov. 27, 2024 — Chronic diseases such as diabetes are on the rise and are costly and challenging to treat. Scientists have discovered a common denominator driving these diverse diseases, which may prove to be a ...

Cooking Up a Breakthrough: Engineers Refine Lipid Nanoparticles for Better mRNA Therapies

Nov. 22, 2024 — Engineers have cooked up a new way to improve mRNA delivery, developing an optimal 'recipe' for ionizable lipids -- key ingredients in lipid nanoparticles (LNPs), the molecules behind the ...

HIV Latency Reversing Properties in African Plant

Nov. 19, 2024 — The Wistar Institute and the University of Buea in Cameroon has uncovered the mechanisms for a medicinal plant with anti-HIV potential in Croton oligandrus Pierre & Hutch, a species of African ...

A New Technology to Isolate Immunostimulatory Members of the Human Gut Microbiota: Next-Generation IgA-Seq

Nov. 14, 2024 — Scientists have developed a new technology to efficiently isolate a specific subset of gut bacteria from fecal samples that are recognized by IgA antibodies. These 'IgA-coated' bacteria are ...

An Advance Toward Inhalable mRNA Medications, Vaccines

Nov. 13, 2024 — Most people don't enjoy getting shots for treatments or vaccines. So, researchers are working to create more medicines, such as those made from messenger RNA (mRNA), that can be sprayed and ...

Prevalence of Unrecognized Cognitive Impairment in Socially and Economically Vulnerable Older Adults Is High

Nov. 7, 2024 — One of the first studies to investigate the prevalence of unrecognized cognitive impairment among patients seen at Federally Qualified Health Centers, has found that it is ubiquitous, especially ...

Education, Occupation, and Wealth Affect the Risk of Cognitive Impairment

Nov. 1, 2024 — Socioeconomic factors such as education, occupation, and wealth influence the likelihood of developing cognitive impairment or dementia in later life and whether a person is likely to recover, finds ...

Heart Failure, Atrial Fibrillation and Coronary Heart Disease Linked to Cognitive Impairment

Oct. 10, 2024 — A new American Heart Association scientific statement suggests addressing cardiovascular health earlier in life may reduce the risk of stroke and help preserve thinking and memory later in ...

Gut Hormones Could Hold the Key to Fighting Fatty Liver Disease

Oct. 7, 2024 — Fatty liver disease is a growing global health concern. Proglucagon-derived peptides (PGDPs), including glucagon, GLP-1, and GLP-2, are known to regulate lipid metabolism in the liver. However, the ...

New Therapeutic Target for Cardiac Arrhythmias Emerges

Oct. 4, 2024 — Researchers identified a lipid that is involved in regulating cardiac ion channels, providing insights into possible mechanisms of cardiac arrhythmias in heart failure and a potential pathway for ...

Siloxane Nanoparticles Unlock Precise Organ Targeting for mRNA Therapy

Oct. 1, 2024 — Engineers have discovered a simple and inexpensive means of directing lipid nanoparticles (LNPs), the revolutionary molecules that delivered the COVID-19 vaccines, to target specific tissues, ...