Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
A new gene therapy has been shown to completely eliminate osteoarthritic pain and significantly reduce long-term joint damage in genetically engineered mice. The therapy works by increasing opioid receptors on nerve cells, making them more responsive to naturally occurring painkillers.
Researchers successfully awakened vision cells in blind mice using gene therapy, restoring their visual abilities. The breakthrough has implications for treating common blinding diseases like age-related macular degeneration and diabetic retinopathy.
The Burroughs Wellcome Fund has awarded $14 million to support the first class of physician-scientists through the Career Awards for Medical Scientists program. The recipients will receive career development funding to advance their research and transition into independent academic careers.
Researchers identified a genetic mechanism that permanently shuts down crucial genes in healthy immune system cells, which could be used to target cancer and infection treatments. The discovery was made in normal blood samples and found in a quarter of leukemia samples, highlighting the potential for this mutation as a therapeutic target.
Researchers at Duke University Medical Center found that patients with specific gene variants were less likely to experience cognitive decline after heart surgery. These variants were involved in the inflammatory system and may provide a biological basis for the protective effect observed.
A recent study reveals that mice lacking OCT1 have reduced metformin effects on AMPK phosphorylation and glucose control. Genetic variations in OCT1 in humans also impact metformin uptake, highlighting the importance of personalized treatment approaches.
Researchers at the University of Texas M. D. Anderson Cancer Center have successfully delivered a cancer-suppressing gene into tumors of stage 4 lung cancer patients via an intravenously administered lipid nanoparticle. The gene, FUS1, was found to be active in metastatic non-small cell lung cancer tumors.
VIB researchers have discovered that MYB duplication is associated with T-ALL cases, leading to increased MYB concentrations. This finding opens up possibilities for targeted therapies against this specific group of patients.
A recent NIDA study has identified genes associated with an individual's ability to quit smoking. The research, published in BMC Genetics, found 221 genes that distinguish successful quitters from those who struggled to stop smoking.
A new blood test can rapidly detect levels of radiation exposure, enabling timely treatment for potentially life-saving. The test analyzes thousands of genes to identify distinct genomic signatures reflecting varying radiation doses.
Researchers found that specific genes distinguish smokers who quit from those who struggle with addiction, suggesting a biological basis for success. The study could lead to personalized treatment plans tailored to individual genetic profiles.
Researchers identify PGC-1alpha as a key genetic component and potential therapeutic target for Duchenne muscular dystrophy. Experimental elevation of PGC-1alpha improves DMD symptoms in mouse models, offering new therapeutic promise.
Researchers at the University of Wisconsin-Madison have created ultrathin films composed of DNA and water-soluble polymers that allow controlled release of DNA from surfaces. These films could be used to deliver genetic material for gene therapy, potentially treating conditions such as cardiovascular disease by preventing smooth muscle...
Researchers at Temple University's Sbarro Institute have discovered a small molecule derived from the spacer domain of the tumor-suppressor gene Rb2/p130. The molecule, named Spa310, has been shown to inhibit tumor growth and reduce cell cycle activity in mice with cancer.
A University of Missouri study found that negative news stories about clinical trials deter people from participating in medical research. The study analyzed 216 New York Times and 124 Washington Post articles, revealing a significant correlation between negative coverage and decreased willingness to participate.
Researchers used gene therapy to shut down a key gene responsible for inherited blindness, reducing mutated rhodopsin by 60%, in an effort to develop a treatment for retinitis pigmentosa. The technique may restore vision in affected individuals if successful.
Researchers at the University of Kentucky are exploring a novel gene therapy approach using DNA nanoparticles to deliver proteins beneficial to brain cells. This technology has shown potential in rescuing dormant brain cells, causing them to produce dopamine, and improving symptoms in animal models of Parkinson's disease.
A Phase I clinical trial suggests gene therapy is a safe treatment option for patients with poor circulation and blocked blood vessels in their lower limbs. The study found that almost half of patients reported complete resolution of chronic pain and over a quarter experienced complete healing of chronic wounds after one year.
Researchers found that increasing connexin26 in mice with missing connexin30 restored hearing sensitivity and prevented hair cell death. The discovery suggests a potential drug treatment for congenital deafness, which may replace gene therapy.
Researchers at Albert Einstein College of Medicine have discovered a genetic signature that identifies cases of lymphoma susceptible to new molecular targeted therapy. The study found that tumors with this signature are killed by the new therapy, while those without it are resistant.
Carbon nanotubes successfully deliver RNA fragments that shut off genes for HIV-specific receptors on human T-cells. This approach significantly slows down HIV infection by blocking the virus's entry points.
Researchers at Johns Hopkins Medicine have identified 34 unique genetic variations associated with sporadic ALS, bringing them closer to developing treatments. The study scanned the entire genome of 276 subjects with ALS and found that these genetic variants are more common in individuals with the disease.
A recent MIT study reveals a genetic link between gene mutations and schizophrenia, focusing on the calcineurin system as a potential target for future treatments.
Researchers have made significant breakthroughs with RNAi gene therapy, showing its effectiveness in shutting down viruses that cause diseases such as hepatitis and HIV in mice. With three human trials underway, the technique may be on the verge of widespread use for treating various human diseases.
Researchers have identified several genes linked to nicotine dependence, including CHRNA5 and NRXN1, which play a role in regulating communication between nerve cells. These findings could lead to the development of more effective smoking cessation therapies tailored to an individual's genetic traits.
Scientists have discovered that human proteins evolve slowly due to dual coding regions in their DNA, which slows down the rate of evolution. This knowledge can be used to develop more effective gene therapy techniques and potentially treat genetic disorders.
Researchers at UT Southwestern Medical Center have developed a novel method to activate specific genes using RNA strands, which can help alleviate disease conditions. By introducing tailor-made RNA strands into cells, the researchers can nudging genes into activity, potentially treating diseases such as cancer.
A combination of bone marrow transplantation and gene therapy has greatly lengthened the lives of laboratory mice with Krabbé disease, a rapidly progressing neurodegenerative disorder also found in people. The dual therapy improved motor skills and increased lifespan by more than twice as long as untreated mice.
Researchers have developed a gene chip that can detect genetic causes of jaundice in children and adults, potentially leading to personalized treatment options. The 'jaundice chip' is nearly 100% effective in detecting common mutations associated with inherited liver diseases.
Scientists have discovered that NSAIDs induce the MDA-7/IL-24 gene, which kills cancer cells while sparing healthy ones. This finding could lead to the development of targeted cancer treatments.
Researchers have identified genetic markers that can predict which patients with major depression are more likely to respond to treatment. Polymorphisms in genes related to serotonin and neurogenesis were found to be associated with improved treatment outcomes.
A study published in JCI Journal shows that silencing the PrPc gene suppresses BSE and CJD accumulation, offering a new approach to treat these fatal diseases. The therapy delayed PrPsc accumulation in mice, providing potential hope for individuals with neurodegenerative disorders.
A clinical trial of gene transfer therapy for erectile dysfunction found significant and sustained improvements in patients who received a transfer gene called hMaxi-K. The therapy works by creating additional potassium channels in smooth muscle cells, relaxing the muscle and allowing blood flow required for an erection.
A new gene transfer therapy has shown promising results in treating erectile dysfunction (ED) and may also have potential benefits for overactive bladder, irritable bowel syndrome, and asthma. The therapy, which targets smooth muscle cells, was well-tolerated and safe in a small pilot study of 11 men.
Researchers have successfully tested a gene therapy to combat alpha-1 antitrypsin deficiency, a common hereditary disorder that causes lung and liver disease. The treatment showed no adverse effects and introduced the corrective gene into patients' blood, with some evidence of protein production.
Researchers developed a new formula to design flows that break polymers into specific lengths or withstand certain flows, with potential implications for industries like shipping and oil. This discovery also enables more precise control over the length of DNA strands in genome sequencing.
Researchers at New York University are developing molecular delivery vehicles to transport nucleic acids into cells. Their work aims to silence genes improperly produced through RNA interference, with potential applications in genetic therapies.
Scientists at Stanford University used gene therapy to improve memory and learning in rats under stress, blocking the negative effects of steroids. The experimental technique transforms harmful corticoids into beneficial estrogens, potentially reducing cognitive side effects of steroids.
A study found that intravenous gene therapy protects normal tissue during whole-body radiation, leading to higher survival rates in mice. The therapy, administered before exposure, appeared to prevent the damaging effects of radiation, suggesting it is a viable delivery method for public protection in nuclear emergencies.
Researchers have successfully treated mice with gene therapy, reversing neurological damage and deficits caused by the genetic defect that leads to metachromatic leukodystrophy. The treatment involves using hematopoietic stem progenitor cells genetically modified to express high levels of ARSA protein.
Researchers discovered that GBA2 is necessary for normal sperm function and male fertility in mice. A lack of GBA2 results in abnormal sperm morphology and decreased fertility, similar to a treatment for Gaucher's disease.
An international team of researchers has identified a novel gene mutation linked to Crohn's disease and ulcerative colitis, which may offer a new pathway for tracking the disease process and potential drug treatments.
A study by University College London researchers found that carrying a common variant of the TCF7L2 gene increases the risk of developing diabetes by 50%, similar to being clinically obese. The genetic variant is present in nearly two-thirds of people with type 2 diabetes.
The Phase I trial demonstrated statistically significant clinical efficacy and neuro-imaging results, with patients showing a 25% improvement in Unified Parkinson's Disease Rating Scale scores. PET scan data revealed a significant improvement in brain metabolism on the treated side of the brain compared to the untreated side.
Scientists have mapped a common form of Fuchs corneal dystrophy to chromosome 18, shedding light on its genetic origins. The discovery has implications for developing gene therapies to treat the condition.
Researchers have developed a gene therapy treatment that restored retinal function to near-normal levels and prevented degeneration in cones of mice with Leber congenital amaurosis. The study shows promise for potential human applications, offering new hope for individuals affected by this condition.
A Dutch researcher found that genetic characteristics, such as sweat production and brain receptor genes, can predict which drug works best for each patient. This information may lead to more effective treatment allocation for alcoholics.
A recent study by Dr. Robert Koenekoop and colleagues has identified the CEP290 gene as the most common cause of Leber Congenital Amaurosis (LCA), a form of congenital blindness. The discovery could lead to improved screening and treatment options for affected children.
Children's Hospital of Pittsburgh researchers aim to develop more effective treatments for patients with chronic lung diseases, including cystic fibrosis and idiopathic pulmonary fibrosis. The $12.8 million NIH grant will support the development of new diagnostic methods and therapies.
Researchers developed a gene transfer method using the mutant form of apolipoprotein A-I (Apo A-I Milano) to treat vascular inflammation and plaque buildup. The study found that Apo A-I Milano gene transfer resulted in a 65% reduction in plaque buildup, compared to a 25% decrease with the normal HDL gene.
A new gene expression-based chemical genomic approach has identified potent inhibitors of androgen receptor signaling, including celastrol and gedunin, which may overcome prostate cancer treatment resistance. Additionally, rapamycin has been found to potentially reverse glucocorticoid resistance in childhood acute lymphoblastic leukemia.
Researchers found that piggyBac transposon is five to 10 times better than other circular pieces of DNA at making a home and difference in several mammalian cell lines. This could lead to safer and more efficient gene delivery for therapeutic applications.
Researchers found that increasing leptin production in the hypothalamus helps regulate insulin secretion, keeping blood glucose levels normal. Gene therapy successfully reversed type 2 diabetes in diabetic mice, even after a high-fat diet.
Researchers found that patients with advanced squamous cell carcinoma of the head and neck whose tumor samples over-expressed p53 protein were significantly more likely to respond to Advexin therapy than those with low p53 protein. The study suggests that p53 overexpression may be a predictive biomarker for Advexin efficacy.
A study by Purdue University researchers has identified unusual genetic traits in voles that challenge current scientific understanding. The vole's unique genetic makeup, including its ability to insert DNA into the nucleus, could have important implications for human genetics and gene therapy.
Researchers found that a single genetic assay can predict response to different targeted therapies in gastrointestinal stromal tumors. Patients with exon 9 mutations or wild-type genes may benefit more from Sutent, while those with exon 11 mutations may require longer Gleevec treatment.
Researchers discovered that viruses can infect cells more efficiently by attaching to different carbohydrates on the cell surface. This finding helps explain how flu and other viruses evade the immune system and may be useful for developing gene therapies for cancer and brain diseases.
Researchers at Virginia Tech have designed polymer macromolecules as effective gene transfer agents, overcoming the need for foreign DNA and viruses. The study's findings focus on the structure of these molecules, which can control their ability to transfer genes across cell membranes.
A Scripps Research Institute team developed compounds that reactivated the frataxin gene in blood cells from 13 Friedreich's ataxia patients, with one compound producing full reactivation in 100% of cells tested. The findings offer a potential therapeutic avenue for the disease, which affects 1 in 20,000 people in the US.
This study confirms the existence of immunological synapses, microanatomical structures similar to those of nerve cells, in laboratory rats. The findings settle controversy over their existence and functional significance during antiviral immune responses, paving the way for further research.