Articles tagged with Gene Therapy
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 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 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 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.
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 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.
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.
The International HapMap Project aims to map the locations of representative tag SNPs in DNA samples from diverse populations. The project's results will increase the power and reduce the cost of future genetic association studies, significantly speeding up the discovery of genes involved in common diseases.
Researchers identified three strongly predictive genes - OPAL1, GNB2L1, and IL-10 receptor alpha - that were associated with better outcomes in pediatric ALL patients. These genes may help improve risk classification and outcome prediction for acute leukemia in children.
A study found that patients with high levels of low molecular weight cyclin E have aggressive, invasive breast cancer, and the protein may be a better predictor of patient outcome than current markers. This altered form of cyclin E can induce genetic instability, making tumors unresponsive to treatment.
A study published in Cancer Research found that combining gene therapy and radiation therapy showed significant declines in PSA levels and eliminated cancer in many patients. The novel treatment has shown promise in treating aggressive prostate cancer, but more research is needed before it can be widely adopted.
Researchers successfully transferred and expressed MGMT into relatively few hematopoietic stem cells using a lentivirus vector, enabling gene-corrected cells to repopulate the hematopoietic compartment. This breakthrough has significant implications for human clinical trials of gene therapy in bone marrow transplantation settings.
Researchers found that blocking laminin-8 gene expression significantly reduced glioma cells' ability to invade neighboring tissue. The study supports the hypothesis that laminin-8 is involved in tumor spread and suggests a potential target for intervention strategies.
A recent study found that patients with a genetic subtype of diabetes have a significantly greater response to sulphonylurea drugs compared to those without a defined cause. This discovery highlights the importance of individualizing treatment for patients with type 2 diabetes, depending on their underlying cause.
The University of Pittsburgh has been designated as a cooperative research center to develop treatments for muscle-wasting diseases. The center will focus on gene and stem cell therapies to treat Duchenne muscular dystrophy, the most common and debilitating childhood disease.
Researchers used alginate, a major constituent of brown algae, to block viral leaks and reduce the concentration of toxic protein IL-12 in mice's livers. The solution improved the effectiveness of gene therapy by preventing sudden animal deaths caused by uncontrolled protein expression.
Researchers found that injecting a gene producing insulin-like growth factor-1 (IGF-1) into muscles delayed disease onset by 31 days, preserved nerve cells, and reduced muscle wasting. This study may lead to a new, gene-based treatment for ALS affecting over 30,000 Americans.
Researchers at Johns Hopkins and Salk Institute develop gene therapy that slows progression of amyotrophic lateral sclerosis (ALS), a devastating disease affecting thousands. In experiments with mice, injection of insulin-like growth factor-1 (IGF-1) into muscles extends survival and improves strength.
Researchers at Project A.L.S. have successfully tested a gene therapy approach that delays disease progression and prolongs survival in a mouse model of ALS. The treatment uses an adeno-associated viral vector to express the IGF1 protein, promoting neuronal survival.
Researchers develop novel gene therapy platform using AAV to deliver galanin, a neuroactive peptide that suppresses seizure activity. The treatment reduces sensitivity to focal seizures in laboratory rats, offering hope for treating temporal lobe epilepsy patients.
David Botstein, known as 'The Father of Modern Genetics', was awarded the 2003 Gruber Prize for his groundbreaking work on human genetic mapping and the discovery of transposons in bacteria. He will continue to lead research at Princeton University's Lewis-Sigler Institute for Integrative Genomics.
Researchers developed a gene therapy technique that uses an oncoretrovirus to transfer MGMT into normal bone marrow cells, allowing for the enrichment of healthy cells. The treatment showed promising results in mice with beta-thalassemia, achieving successful in vivo selection in 66% of cases.
Scientists have identified a preference for insertion at the beginning of genes and near actively expressed genes, which may explain why gene therapy patients developed leukemia. The discovery could lead to improved gene therapy techniques that insert genes in less risky areas.
A study by Temple University researchers found that pRb2/p130 binds to the estrogen receptor alpha and recruits molecules that make breast tumors more responsive to treatment. However, mutated pRb2/p130 signals can silence the expression of the estrogen receptor, making therapies less effective against cancer cells.
Weizmann Institute researchers have solved the 3D structure of the glucocerebrosidase enzyme involved in Gaucher disease. The discovery may lead to the design of more effective therapies, including enzyme replacement therapy and small molecule supplements.
Scientists have successfully induced the growth of new sensory hair cells in adult guinea pigs using gene therapy. The Math1 gene was inserted into non-sensory epithelial cells lining the inner ear, leading to the formation of new hair cells and attracting the growth of new fibers from auditory neurons.
Researchers discovered mutations in 14 tyrosine kinase genes, which play key roles in controlling cell growth and differentiation. These findings open the door to individualized treatment approaches for colorectal cancer, allowing for personalized therapeutics tailored to each patient's tumor DNA.
Researchers at Brenner Children's Hospital have developed a new gene therapy technology that uses highly efficient protein delivery to correct mitochondrial defects. This approach has the potential to prevent or treat genetic diseases like SIDS and TFP deficiency in infants, as well as heart and brain diseases later in life.
Researchers successfully integrated genes into hematopoietic stem cells to produce normal red blood cells in mice with beta-thalassemia and sickle cell disease. The technique uses a non-pathogenic AIDS virus gene to ferry DNA into the cells, selectively enriching genetically modified stem cells.
Scientists create hybrid virus that can attach to lung cells' top surface, allowing healthy genes to enter and correct genetic defects associated with cystic fibrosis. The new approach increases production of viral particles, a crucial step towards preclinical studies.
A recent Phase I study of compacted DNA gene therapy for cystic fibrosis demonstrated safety and tolerability. The treatment resulted in a meaningful increase in chloride ion transport in the nose, suggesting successful gene transfer and potential therapeutic benefits.
Researchers have discovered that injecting herpes simplex virus vectors can deliver neurotrophic factors to damaged nerves, improving intracavernous pressure in rats. This concept could translate to humans, enabling gene therapy as a prophylactic treatment for men undergoing radical prostatectomy.
The BP1 gene is found to be active in 57% of Caucasian women and 89% of African American women with breast cancer. Gene therapy targeting BP1 may help treat non-hereditary breast cancer, which accounts for 95% of cases.