A new software called Notung allows researchers to identify genes that arose recently and understand their role in modern organisms. The tool uses evolutionary scenarios and gene duplications to provide insights into gene evolution and function.
A breakthrough in gene therapy has successfully corrected hemophilia in laboratory animals by producing a normal level of clotting factor activity and preventing bleeding incidents.
A recent study reveals that cognitive therapy is as effective as antidepressant medication in treating depression, but with a lasting impact even after treatment concludes. Cognitive therapy equips patients with skills to manage problems and emotions, outperforming medication in preventing relapses over time.
Researchers at UT Southwestern Medical Center have developed a new gene therapy technique that uses homologous recombination to replace mutated genes in human immune cells, restoring both gene function and protein production. This approach has shown promising results in treating severe combined immunodeficiency disease (SCID) and may a...
Researchers at Duke University Medical Center have successfully reversed glycogen buildup in heart and skeletal muscle of genetically altered mice using a muscle-targeted gene therapy. This approach shows promise as a potential treatment strategy for Pompe disease patients who fail to respond to enzyme replacement therapy.
Researchers used RNA interference to silence mutated SOD1 genes in ALS mice, reducing disease progression and improving neuromuscular function. This breakthrough suggests gene silencing as a potential therapy for incurable progressive neurological diseases like ALS and Parkinson's.
A new tumor-suppressor gene called mats has been discovered by researchers at Penn State, which works with another tumor suppressor to control tissue growth. The study found that the mats gene can shrink tumors in flies and may regulate cell number and tissue growth by restricting cell proliferation.
A meta-analysis of nine randomized trials found no significant difference in death rates or disease progression between neoadjuvant and adjuvant systemic therapy. However, neoadjuvant therapy was associated with a higher risk of loco-regional disease recurrence when radiation therapy was used without surgery.
A new study has provided proof of principle for a combined stem cell-gene therapy approach as a potential treatment for cystic fibrosis. The researchers were able to correct the CFTR gene defect using adult stem cells and ex vivo gene therapy, which may offer advantages in screening and long-term function.
Researchers develop gene therapy to treat XP, a devastating disease causing cancerous lesions on exposed skin. The treatment restores DNA repair mechanism, preventing cancerous growth and sunburn-like symptoms in mice.
Researchers at Thomas Jefferson University used gene therapy to restore heart function in rats with failing hearts, reversing cardiac dysfunction. The study found that delivering the human S100A1 gene improved heart muscle efficiency and reversed cardiac dysfunction.
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 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.
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.
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 randomized clinical trial found that combining six months of hormone therapy with radiation therapy reduced the risk of death from prostate cancer by two-fold. Patients treated with this combination therapy had a lower risk of requiring salvage hormone therapy in the long term.
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 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 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.
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 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 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.
Researchers developed a new method to alter gene expression levels without disrupting essential control elements. This technique uses the 3' untranslated region (UTR) to influence protein production, allowing for predictable and controlled changes in gene expression.
Researchers from the University of Texas M. D. Anderson Cancer Center have developed a novel approach to gene therapy that harnesses mesenchymal progenitor cells (MSC) to target cancer metastasis. The strategy has shown promising results in animal studies, with MSC gene therapy curing up to 70% of mice implanted with human ovarian cancer.
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 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.
Researchers are testing a gene therapy approach called tumor immunization, which involves delivering the IL12 gene to cancer cells to trigger an immune response. The method has shown promise in laboratory and animal studies, with up to 20% of animals experiencing complete tumor eradication.
Recent evidence from randomized trials on hormone therapy and hypertension treatment led to a decline in hormone therapy prescriptions following publication of trial results. Alpha-blocker prescriptions also declined after the Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial (ALLHAT) early termination.
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.
Two independent studies successfully selected and expanded gene-corrected human stem cells using a novel drug-resistance gene. This breakthrough may be useful in human clinical trials of gene therapy for bone marrow transplantation settings. Additionally, upregulation of Irs2 promotes beta cell growth, survival, and insulin secretion, ...
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 at three new NIH-funded centers are working on developing gene and stem cell therapies to treat Duchenne muscular dystrophy. The centers will study various aspects of gene therapy, including the delivery and engraftment of muscle stem cells into diseased heart tissue.
Researchers found that the HIV vpr gene exploits the normal repair process of cells to stop vital white blood cells from replicating, thus disabling the immune system. The study suggests a possible treatment for AIDS-related immune-system damage using medicines that prevent the human ATR gene from being activated by HIV's vpr gene.
A study examined whether limited-field radiation therapy could be an effective alternative to whole-breast radiation therapy for patients at low risk of recurrence. The results showed no significant difference in local recurrence rates or overall survival between the two groups after five years of follow-up.
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.
Scientists have successfully silenced mutant genes without affecting normal gene copies using RNA interference, a promising approach for treating diseases like Machado-Joseph disease, Huntington's, and Alzheimer's. This breakthrough technique has the potential to selectively turn off disease-causing genes, preserving essential normal g...
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.
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.
Researchers at Baylor College of Medicine have made a breakthrough in developing a gene therapy that can cure diabetes in mice. The treatment uses the NeuroD gene to induce liver cells to produce insulin and other hormones associated with the pancreas' endocrine system.
The Alliance has awarded grants to Dr. Andrew Davidoff, Dr. Thomas Griffith, and Dr. Jeffrey Bartlett to develop gene therapies targeting neuroblastoma, prostate cancer, and ovarian cancer. The research holds promise for improved quality of life and reduced side effects for cancer patients.
Researchers have identified a new gene, endothelial lipase (LIPG), that regulates HDL cholesterol levels and impacts the risk of developing heart disease. By altering LIPG expression in genetic models, scientists found an inverse relationship between HDL cholesterol and EL expression.
Scientists have achieved germline transmission of 'gene knockdown' in mice by using genetic engineering to create mouse embryonic stem cells targeted with RNAi. This enables the manipulation of gene activity in specific tissues and allows for switching on and off at any time during development or adulthood.
Researchers discovered a gene mutation associated with a form of Charcot-Marie-Tooth disease, affecting approximately 1 in 2,000 people. The LITAF gene mutation was found in families with a history of the disease, suggesting its potential as a molecular marker for diagnosis.
Researchers have discovered that the FoxM1B gene plays a crucial role in tissue healing and regeneration. The study found that the gene is essential for cells to divide and multiply, allowing tissues to repair and regenerate. Without FoxM1B, DNA duplication fails, leading to accelerated aging and age-related diseases.
USC researchers have successfully used gene therapy to prompt mouse cells to produce human collagen, a crucial step towards treating patients with dystrophic epidermolysis bullosa. This breakthrough could lead to the development of new treatments for this debilitating skin condition, which causes blisters and permanent scars.
Researchers at Vanderbilt University Medical Center have developed a novel gene therapy approach that repairs messenger RNA, which could lead to effective treatments for inherited diseases. The method uses ribozymes to correct defective genes and has shown promising results in animal models.
A new gene therapy approach has been shown to virtually eliminate cancerous tumors in mice by boosting their immune systems. The treatment, which blocks a key immunosuppressor called TGF-beta, resulted in 70-80% survival rates in mice with melanoma and prostate cancer.
Researchers at UVA have identified a gene called RhoGDI2 that plays a crucial role in preventing the spread of cancer. The study found that replacing this gene in human metastatic cancer cells can suppress their ability to metastasize, suggesting new therapeutic options for treating metastatic disease.
A study found that an uncommon variation of the Nogo gene increases schizophrenia risk, particularly when inherited from both parents. One in five people with schizophrenia has this risk gene, and researchers hope to discover new related genes to aid diagnosis and treatment.
Researchers have developed a novel system of gene therapy that targets lung stem cells to treat cystic fibrosis. By using a natural compound found in the lungs, the therapy enables airway cells to take up the therapeutic gene and shows substantial recovery from the disease defect.
Researchers have developed a gene therapy technique that integrates DNA without using viral vectors, inserting the DNA at known locations. The technique successfully integrated a gene coding for Factor IX into mouse DNA, making 12 times more protein than control mice.
A new study using gene therapy has shown promise in treating Parkinson's disease by 'resetting' overactive brain cells. The therapy may arrest or delay disease progression, offering a safer and more powerful alternative to current treatments.
Researchers discovered a gene, Hsp27, that protects mature nerve cells from death following injury. Delivering this gene via therapy may lead to new treatments for neurodegenerative diseases and nerve injuries.
Researchers have successfully delivered the full-length dystrophin gene to mice with muscular dystrophy using stripped-down vectors, restoring normal muscle function. The breakthrough could pave the way for human clinical trials to assess the safety of this method in patients.
A novel gene therapy technique developed by Stanford researchers allows for the integration of large genes into human chromosomes, enabling continuous production of proteins. This technique has the potential to treat a variety of diseases, including Duchenne's muscular dystrophy and cystic fibrosis.
Researchers have developed a gene therapy approach to treat mucopolysaccharidosis VII, a disorder affecting multiple organ systems, in dogs. The treatment involves four intravenous injections of a retroviral vector expressing canine beta-glucuronidase, resulting in normal enzyme activity and near-normal mobility in treated dogs.
Researchers developed a gene therapy combining radiation therapy and cisplatin to target tumor cells, resulting in significant regression with no additional toxicity. The treatment uses a modified cold virus to insert the TNF gene into tumor cells, enhancing anti-cancer effects.
Researchers at Stanford University Medical Center have developed a gene-therapy technique that can switch off genes in mice using RNA inhibition. The method has shown promising results in reducing light production from a firefly gene, suggesting its potential use in treating diseases such as cancer and hepatitis C by deactivating virus...