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 technique has successfully treated irregular heart rhythms in pigs, using a 'painting' method to deliver the therapy. The technique, which uses a plastic, gel-like substance to contain the gene therapy, shows promise for treating atrial fibrillation, a common heart condition.
Researchers at the University of Pennsylvania School of Veterinary Medicine have identified key growth factors essential for sustaining spermatogonial stem cells in culture. By understanding these factors, scientists hope to develop new fertility therapies and create sperm in vitro.
Researchers at Pitt University successfully reversed peripheral neuropathy in diabetic mice using gene therapy based on HSV vectors, delivering neurotrophic factors to affected neurons. The approach restores lost nerve endings and prevents progression of neuropathy, with promising results for potential human treatment.
Children's Hospital Boston geneticist Dr. Kunkel has made significant contributions to understanding muscular dystrophy, from identifying genes to developing new therapies. His work aims to find patterns common to all muscular dystrophies and those distinct to specific forms of the disease.
A recent study found that demethylation agents not only restart cancer-suppressing genes but also knock methyl groups off other genes, turning hundreds more off. The research raises concerns about the impact of these agents on gene expression and their potential to exacerbate cancer development.
Researchers at St. Jude Children's Research Hospital developed a gene vector that allows hematopoietic stem cells to produce fetal hemoglobin, reversing beta-thalassemia in mice. The technique uses a new vector with added regulatory elements to improve the expression of the gamma-globin gene.
Researchers have identified a new region on the bcl-2 gene that regulates its expression and stability. This discovery may lead to the development of new cancer therapies by targeting the CA-repeated Region (CAR) to reduce bcl-2 levels in cancer cells.
NIST has awarded up to $80.1 million in funding for 32 new advanced technology program (ATP) projects, spanning various key technology areas including energy, medical diagnostics, electronics, and computer interfaces. The projects aim to improve productivity, facilitate trade, and enhance quality of life.
Researchers have developed a novel gene regulation strategy using ribozymes, which can be controlled with virtually any drug, offering a safer alternative to existing methods. The technique enables the easy turn on and off of genes, allowing for potential applications in therapeutic and research settings.
A study funded by the Christopher Reeve Paralysis Foundation has identified novel potential therapeutic targets for spinal cord repair using microarray technology. The study analyzed 108 genes and found patterns of gene expression that could help researchers identify new treatments.
A study has identified two distinct subclasses of liver cancer with differing survival rates, providing new molecular insight into the pathogenesis of hepatocellular carcinoma. The findings suggest that gene expression profiles can accurately predict patient survival and may lead to the development of more targeted therapies.
Researchers will study 120 patients with chronic and aggressive periodontitis to identify molecular basis of disease and develop targeted therapies. The goal is to reduce risk of periodontitis and improve treatment outcomes for the leading cause of adult tooth loss.
A recent study found that genetic variations in the HLA and TNF gene regions are associated with response to treatment in early rheumatoid arthritis patients. Patients with two copies of the shared epitope were four times more likely to achieve significant improvement in disease activity compared to those with one or no copies.
Scientists uncover vital role of Periaxin gene in causing CMT, a disabling disease affecting 23,000 people in the UK. The new research provides insight into how nerves work and may lead to development of gene therapies to correct faulty genes.
Researchers developed a novel gene-based therapy that substantially decreased tumor size and eliminated cancer cells in experimental mice. The treatment, which uses recombinant adenoviruses encoding interferon-alpha, showed promise in responding to even resistant bladder cancer cell lines.
A gene associated with lupus has been identified, suggesting a potential breakthrough in diagnosing and treating the disease. The discovery could lead to earlier intervention and more effective therapies.
Researchers found that different retroviral vectors from ASLV, HIV, and MLV integrate into human chromosomes at varying sites. The study suggests ASLV may be a promising candidate for human gene therapy due to its distinct integration preferences.
Researchers at Jefferson Lab are adapting detector technology to develop a new gene therapy technique for cystic fibrosis, which affects 30,000 Americans. The technology allows for successful imaging of gene transfer in mice, paving the way for clinical trials.
Researchers at UCSD and VA San Diego Healthcare System have developed a novel method of transcriptional gene silencing in the nucleus, utilizing short interfering RNA (siRNA). This approach may provide a long-lasting solution for gene regulation, with potential applications in treating diseases such as cancer and HIV.
Researchers have developed a novel gene therapy that combines a therapeutic gene with a genetic sensor to protect the heart from damage caused by reduced blood flow. The therapy, which was tested in rats, showed a significant reduction in tissue death and damage compared to untreated animals.
Researchers have identified nearly 100 genes linked to cancer, shedding light on the processes driving disease progression. The study highlights the potential of targeted therapies and early detection methods to manage cancer.
Researchers have successfully delivered a mini-dystrophin gene to all skeletal and cardiac muscles of an adult mouse with muscular dystrophy, reversing the disease. The breakthrough uses a safe and simple method to target muscle cells without triggering an immune response.
Researchers found genetic mutations in fibulin genes, specifically FBLN5, that could contribute to AMD. However, these changes were not statistically significant, and the study highlights the importance of precise search methods for genetic causes.
A LSU vet school professor has successfully treated a dog with cancerous tumors using gene therapy, which helps the body develop an anti-tumor memory. The treatment involves electroporation and cytokine gene therapy, resulting in the elimination of smaller tumors and reducing the size of larger ones.
OHSU researchers are studying vaccination methods for seniors and children, with a focus on developing protection against infectious diseases. The study aims to understand immune system changes that occur with aging and develop methods to manipulate the system for better protection.
Using guinea pigs, researchers increased production of G-protein Gem, which decreased calcium current densities by 30-90% and helped steady irregular heartbeats. Gene therapy shows promise as an alternative to calcium channel blockers with fewer side effects.
Researchers at UNC have developed a high-throughput technique to rapidly analyze gene functions, resolving two bottlenecks in the process. This method has the potential to accelerate the development of new drugs by quickly identifying and testing candidate genes.
Scientists used gene therapy to deliver RNA that silenced the disease-causing SCA1 gene in mice with spinocerebellar ataxia 1, preventing neurodegeneration. The approach also protected brain cells from destruction and prevented protein clump buildup.
Research shows that children who survive ALL have a higher rate of genetic mutations, which can lead to increased risk of developing secondary malignant neoplasms and other complications. The study found that the number of genetic mutations accumulated over the course of treatment and remained elevated after completion.
A study found two common genetic polymorphisms associated with reduced statin effectiveness, affecting total and LDL cholesterol levels. The findings suggest personalized medicine and genetic screening could improve treatment outcomes.
Researchers have developed a gene therapy that protects bone marrow cells from chemotherapy's debilitating effects. The study found up to 41% transfer of the protective gene to blood stem cells in patients with advanced cancer, offering new hope for improved treatments.
Researchers found that women with a specific gene mutation in the CYP2D6 gene had lower levels of endoxifen, a byproduct of tamoxifen metabolism. This suggests that the gene mutation may affect the potency of tamoxifen when combined with certain antidepressants like paroxetine and sertraline.
Researchers developed a regulatable gene therapy that can be turned on and off using doxycycline, offering potential for treating Parkinson's disease. The treatment uses the glial cell line-derived neurotrophic factor (GDNF) protein to promote survival and differentiation of dopamine neurons.
Researchers at the University of Wisconsin-Madison have developed a groundbreaking gene therapy technique that safely delivers therapeutic DNA to muscle cells. The innovative approach, which uses a non-viral method to inject genes into limb veins, has exciting implications for treating muscle and blood vessel disorders.
Researchers have discovered that a gene therapy using growth factor VEGF can slow down the onset and progression of ALS in mice, increasing life expectancy by 30%. This treatment has shown promising results without toxic side effects, offering new hope for patients with this incurable disease.
Researchers from USC provide a new perspective on treating genetic disorders by switching genes back on using epigenetic therapy. Several chemical compounds have been found to affect epigenetic gene changes and are being tested in clinical trials, including one recently approved for myelodysplastic syndrome.
A gene mutation may cause misrouting of proteins in retinal cells, leading to macular degeneration. Research aims to understand the biological function of the gene and its role in fatty acid metabolism.
Researchers used gene therapy to increase dopamine D2 receptor levels, reducing preference for alcohol and consumption by half in genetically predisposed rats. The treatment showed significant reductions in drinking preference and consumption in both groups, with the greatest effects observed within days of treatment.
Researchers at UCLA successfully deliver gene therapy agents directly into tumor cells with minimal complications, improving effectiveness and reducing side effects of existing treatments. The study demonstrates the feasibility and safety of CT-guided injection for gene therapy delivery.
A study of 160 pregnant women with genetic risk factors for thrombophilia found that enoxaparin significantly reduced pregnancy loss compared to low-dose aspirin. Women taking enoxaparin had a normal live birth rate of 86% and healthy newborns, while those on aspirin suffered more pregnancy losses.
A new gene-based screen developed by Stanford researchers can accurately predict a person's response to treatment and identify patients who may benefit from novel therapies. The screen uses a technique called RT-PCR to analyze genes associated with cancer survival, offering a more accessible alternative to existing microarray technology.
A study by Johns Hopkins Medicine found that a gene defect, PASG, is linked to premature aging in mice. The researchers discovered that the mutated gene causes cells to age and die prematurely, leading to growth problems and early death.
Recent studies have identified key molecular markers of cognitive decline in Alzheimer's disease, revealing a complex role of amyloid proteins in the condition. These findings have significant implications for developing new treatments that target the underlying processes driving the disease.
Researchers have identified a novel gene linked to familial Parkinson's disease, revealing a new pathway for therapeutic treatment. The discovery places mitochondrial function at the centre of Parkinson's research, particularly in relation to oxidative stress and brain cell degeneration.
Scientists have engineered a vaccine vector that targets mucosal surfaces, which are entry points for many pathogens. The hybrid vector combines features of adenovirus and reovirus to repel viruses like HIV-1 and infectious bioweapons.
Genetic studies of leukemia patients have led to the identification of specific gene mutations linked to treatment responses and drug resistance. This information is guiding the development of new drugs targeting specific cell molecules, reducing toxic side effects.
Researchers at the University of South Florida have developed a biosensor-regulated gene therapy that protects heart muscle cells from further injury after a heart attack. The therapy uses an oxygen-sensitive switch to turn on protective genes, which were shown to limit tissue scarring and improve heart function in mice.
Researchers at St. Jude Children's Research Hospital developed a genetic trick adapted from viruses to create gene therapy vectors that can efficiently produce multiple proteins in specific amounts. This technique uses self-cleaving peptides to break down long protein complexes into smaller, functional proteins.
Researchers used gene expression analysis to measure the activity of thousands of genes in adult T cell acute lymphocytic leukemia (T-ALL) patients. They identified a single gene, IL-8, that was highly expressed in resistant patients and found a group of 30 genes associated with complete remissions.
Researchers identified gene expression profiles associated with short-term and long-term outcomes in adult T-ALL patients. A three-gene model was found to be highly predictive of remission duration, offering new hope for customized treatment plans.
Researchers discovered a link between the Endothelial NO Synthase gene and Alevolar Capillary Dysplasia (ACD), a fatal lung disease affecting newborns. The study found that mice lacking this gene developed major defects in lung development, mirroring human cases of ACD.
A University of Iowa study suggests that blocking the action of a certain protein involved in inflammation might be beneficial for organ transplantation, heart attacks, and possibly stroke. The study found that selective inhibition of this protein can prevent tissue damage caused by ischemia/reperfusion injury.
Researchers developed gene therapy targeting phospholamban, a protein contributing to heart failure. Studies in rats showed improved heart function and reduced scar tissue formation after treatment.
A study by James Carrington and Steve Jacobsen reveals distinct classes of small RNAs in plants with specialized functions. These include genome maintenance, regulation of specific genes, and defense mechanisms, shedding light on the evolution of RNA-mediated gene silencing.
Researchers found that viruses used for gene delivery can influence gene expression, with AAV affecting genes minimally and adenovirus triggering broader responses. The study provides a systematic explanation for the relative safety profiles of two commonly used gene therapy vector classes.
Researchers developed a novel tumor cell-specific therapy that restores p53 protein function in cancer cells. This approach eliminates tumors and increases animal survival by up to six times, offering promising implications for targeted therapies.
Research reveals that specialized teaching can give dyslexic brains a 'jump start,' activating key areas used by normal readers. Training the meaning word form and sounds improves ability to analyze sounds and words in specific brain regions.
Researchers have developed a novel method for gene delivery using steroid-coated DNA, which improves cell uptake and reduces inflammatory immune responses. The technique has the potential to enhance gene transfer in humans, especially in inflamatory diseases.
A Stanford team has developed a simplified method for generating siRNA molecules to disable genes, overcoming the technique's limitations in expense and labor. The new protocol allows researchers to create libraries of siRNA molecules for all known genes, enabling the identification of genes that play critical roles in stem cell function.
Researchers found a correlation between genetic variations in the VEGF gene and psoriasis susceptibility. This discovery may lead to targeted therapies blocking the activity of VEGF, which could provide relief for patients.