Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
Researchers have successfully used gene therapy to treat two young children with ADA-SCID, a rare form of SCID that requires regular injections of the bovine form of ADA enzyme. The new method involves removing bone marrow cells and engineering them to produce healthy immune cells, offering a potentially lower-risk alternative to bone ...
Biologists found that nematodes use a sophisticated mechanism to render transposons harmless, preventing them from making proteins and jumping through DNA.
Researchers at Georgetown University Medical Center have developed a germ cell line derived from spermatogonia, offering a basic tool for studying male infertility and testicular cancer. This cell line exhibits characteristics of normal adult spermatogonial stem cells and differentiates into advanced germ cell types upon stimulation.
Researchers created a mouse model of hair loss syndrome, which sheds light on the complex interactions between genes and their effects on human diseases. The study found that genetic background plays a significant role in determining the severity of the condition, and could potentially inform gene therapy approaches.
A recent study, TRAFFIC, found that intra-arterial infusions of recombinant fibroblast growth factor-2 (rFGF-2) improved exercise capacity in patients with moderate-to-severe intermittent claudication. Peak walking time increased by 1.77 minutes with single-dose rFGF-2 and 1.54 minutes with double-dose rFGF-2.
A new gene therapy tool has shown promising results in treating mice with hemophilia A by correcting the faulty factor VIII gene. The technology uses RNA trans-splicing to produce normal factor VIII protein, offering a potential cure for this genetic disorder.
Dr. Katherine High, a leading gene therapy researcher, has been chosen by the Howard Hughes Medical Institute for her groundbreaking work on treating hemophilia B. Her research holds promise for delivering therapeutic genes directly into patients' cells to improve blood clotting and quality of life.
Researchers have discovered the location of the genes that can cause heart attack, a breakthrough that could lead to new means of prevention and treatment. The study used genetics maps to scan chromosomes and found regions on chromosomes two, three, and 20 linked with heart attacks.
Scientists have found a way to activate the utrophin gene, which can help compensate for the lack of dystrophin protein in muscles affected by Duchenne's muscular dystrophy. The study suggests that over-producing utrophin may be a viable alternative to adding a working copy of the dystrophin gene through gene therapy.
Research findings link insulin-like growth factor I (IGF-I) to decreased lung cancer risk when bound by IGFBP-3. Environmental factors influence DNA methylation patterns in tumor suppressor genes, which may impact cancer incidence. Electro-gene therapy enhances IL-12 protein delivery, triggering long-lasting antitumor responses.
Researchers have successfully used a virus that causes warts to restore LDL receptor function in cells with defective genes, offering new hope for treating familial hypercholesterolemia. The next step is testing this technique in animals and humans to determine its safety and effectiveness.
Researchers at UT Southwestern and M.D. Anderson have identified eight genes on chromosome 3 that act as tumor suppressors in lung cancer. These genes were found to inhibit tumor growth and induce cell death in human lung cancer cells, suggesting a potential new approach for treating the disease.
A new type of dendritic cell has been found to prevent diabetes in mice by causing T cells to die rapidly. In a study, researchers treated mice with this special dendritic cell and found that half remained diabetes-free at one year.
Researchers analyzed participants' CETP genes to find genetic markers for heart disease. They discovered a pattern of gene variations called haplotype, which showed a significant association with the person's response to statin therapy.
Researchers discovered two mechanisms by which adenoviruses establish persistent infections: interfering with apoptosis signaling and preventing apoptosis through the Receptor Internalization and Degradation complex. This knowledge could improve the usefulness of adenovirus vectors in gene therapy.
The Lancet Neurology aims to report the most important advances in neurology, predicting risk of neurological conditions through genetics and imaging. Experimental therapies like gene therapy and stem cells offer potential for treating neurological disorders.
A study by UC Berkeley's Bruce N. Ames found that megavitamin therapies can effectively treat over 50 genetic diseases, mostly rare metabolic disorders due to defective enzymes. High-dose vitamins, particularly B vitamins like niacin and thiamine, may also improve metabolism in older populations.
A team of researchers at the University of Pittsburgh has discovered a gene mutation responsible for hereditary gingival fibromatosis (HGF), a rare condition characterized by gum overgrowth. The discovery may lead to new therapies, including tissue engineering, and could have implications for treating common dental problems like period...
Researchers successfully treated rats with chemical-induced Parkinson's disease using gene therapy, restoring normal limb movement. The treatment involves delivering two corrective genes into a specific brain region to prime the production of dopamine, which coordinates limb movements.
Researchers found that women with a common mutation in the estrogen receptor alpha gene had dramatic increases in high-density lipoprotein (HDL), or the 'good' cholesterol. This discovery suggests that genetic testing may help doctors make better decisions about hormone replacement therapy for preventing heart disease.
Researchers have discovered at least 100 genes that show significant differences in activity between non-invasive ductal carcinoma in situ (DCIS) and invasive breast cancer (IBC). The study's findings suggest that defects in these genes may play a role in the progression from non-invasive to invasive cancer.
A new intracoronary gene therapy has shown promising results in treating stable angina, a condition affecting nearly 7 million people nationwide. The study, led by the University of Vermont, involved 79 patients and demonstrated improved exercise ability compared to placebo groups.
Researchers successfully combined therapeutic cloning, embryonic stem cell differentiation, and gene therapy to treat a genetic immune disorder in mice. The study demonstrates the potential for nuclear transplantation therapy to correct genetic mutations and restore function in human patients.
Researchers have identified key genes associated with cardiac hypertrophy, hypertension, and dilated cardiomyopathy. Genetic testing is also being developed to predict susceptibility to coronary artery disease, offering new avenues for preventive medicine.
Gene therapy has shown promising results in hemophilia trials, with patients experiencing significant improvements in quality of life. The treatment involves inserting a normal version of the defective gene to produce clotting factor, and has been tested in small animals before being moved to human trials.
Researchers at Northwestern University have developed a gene therapy that reduces inflammatory cytokines involved in joint inflammation. The treatment, which uses the IL-13 gene, has been shown to suppress joint inflammation and prevent the development of arthritis in rat models.
Scientists have identified the PKHD1 gene as the cause of autosomal recessive polycystic kidney disease (ARPKD), a life-threatening genetic disorder. The discovery provides a genetic roadmap for understanding how the disease progresses, paving the way for more accurate diagnoses and treatments.
Researchers have developed a gene therapy using human blood outgrowth endothelial cells to treat hemophilia A. The treatment, which has shown long-term attainment of robust levels of factor VIII, is promising and may pave the way for testing in humans.
A researcher at Medical College of Georgia has identified three genetic flaws on chromosomes in patients with the most common type of Alzheimer's disease. Dr. Poduslo's study suggests that late-onset Alzheimer's is linked to multiple genes and may be subdivided into specific categories, leading to improved diagnosis and treatment options.
Researchers used Sangamo's ZFP technology to repress a specific gene variant, preventing fat cells from developing. This breakthrough demonstrates the precision of ZFPs in regulating gene expression and may lead to new treatments for diseases related to fat cell development.
The American Society for Neuroregeneration and Transplantation (ASNTR) has published guidelines to ensure the development of novel cell and gene replacement therapies for brain disorders. The recommendations focus on preliminary study design, clinical trial methodology, and conflict of interest disclosure.
A new gene therapy method has successfully corrected sickle cell disease in mice by transferring an anti-sickling gene to bone marrow, preventing the formation of deformed red blood cells. The therapy, developed using a viral delivery system, resulted in up to 99% expression of the new gene in circulating red blood cells.
The UNC-Chapel Hill Gene Therapy Center will use the funds to translate basic research knowledge into safe human clinical trials, with a focus on cystic fibrosis and hemophilia. The center aims to provide new treatments for diseases caused by single-gene defects.
Scientists at Uppsala University have identified a gene that regulates mast cell survival upon allergic activation, providing hope for a novel therapy to treat allergies. Inhibition of this gene, A1, leads to the death of mast cells, decreasing their numbers and alleviating allergic symptoms.
Researchers led by Robert E. Braun analyzed gene expression after adenovirus delivery using both in vitro and in vivo studies. The study found no infection of reproductive stem cells, supporting the safety of adenovector use in cardiovascular therapy.
Researchers genetically engineered mice to overexpress the HIF-1α gene in skin cells, resulting in a 70% increase in capillaries and normal-like blood vessels. The study suggests that HIF-1α harnesses VEGF expression in a way that could be beneficial therapeutically for conditions like ischemic tissue and recalcitrant wounds.
Researchers at Women's and Children's Hospital in Adelaide have developed a way to safely use Human Immunodeficiency Virus Type 1 (HIV-1) to transfer therapeutic genes into human cells. This method has wide applicability for various human genetic diseases and is currently being tested on animal models before moving to human trials.
A systematic study confirms the potential of pharmacogenomics to improve drug therapies by selecting treatments based on a patient's genetic profile. However, the practice faces significant challenges, including inadequate training, funding, and concerns about racial inequities.
In two studies, VEGF gene therapy significantly reduced angina episodes and improved exercise tolerance in patients with end-stage coronary heart disease. The treatment has shown impressive clinical results, with most patients experiencing continued improvement in daily life after treatment.
A new 'double suicide gene' therapy has shown promise in treating prostate cancer by carrying pairs of fused genes directly into cancer cells and inducing self-destruction. The technique achieved "greater levels of targeted cytotoxicity" than single suicide gene therapy, with minimal toxicities reported among patients.
Recent research highlights the significance of sodium-calcium exchange in various physiological processes, including vision, secretion, and cardiac contractility. The study also explores its potential applications in cancer therapy and heart function.
Researchers at Brookhaven National Laboratory discovered that coxsackievirus forms pairs on the surface of human cells, increasing the likelihood of infection. The study reveals hidden binding sites on the virus that evade the immune system, making it hard to defeat.
Researchers have found that a gene therapy technique can reduce alcohol consumption in rats by increasing dopamine receptor levels. This approach may offer a new potential treatment for human alcohol abuse.
Researchers at UC Berkeley have discovered a single gene that can stimulate the immune system to reject a range of cancer tumors. The gene, which activates natural killer cells and other immune cells, shows promise as a potential basis for a vaccine therapy.
Researchers have successfully used gene therapy to reduce new blood vessel growth by up to 90% in mice with conditions similar to macular degeneration and diabetic retinopathy. Two genes, endostatin and pigment epithelium-derived factor (PEDF), were injected into the animals' eyes or tails to demonstrate the potential of gene therapy a...
Researchers developed a gene therapy that increased D2 receptor levels in rats, leading to a significant drop in alcohol intake. The treatment showed promise in reducing drinking preference and behavior in both groups of rats.
Researchers at UCSD will team up with Celera Genomics to identify and genotypic genetic variations in patients with hypertension, aiming to develop individualized treatment plans based on genetic profiles. The goal is to predict therapeutic drug responses and minimize side effects.
Researchers used gene therapy to treat mice with beta-glucuronidase deficiency, a condition that affects approximately 1 in 5,000 babies. The treatment showed promise by improving symptoms for up to a year, but six mice eventually developed cancer, raising questions about the long-term safety of this approach.
Scientists have produced new adult neurons from precursor cells using a growth factor, which could potentially treat neurological diseases. The technique, supported by the NIH, involves introducing brain-derived neurotrophic factor (BDNF) into the adult rat brain.
Scientists have identified an important parallel between C. elegans and human NMD pathways, revealing a potential therapeutic strategy for masking genetic mutations. Inactivation of the human homolog of the C. elegans smg-1 gene inhibits NMD, allowing truncated proteins to accumulate and potentially compensate for genetic disorders.
Research reveals heritability plays a major role in determining the age of menopause, with significant implications for women delaying childbirth beyond 30. Genetic analysis suggests that a woman's store of eggs declines throughout life, triggering menopause.
The complete genome of pneumococcus has been sequenced, revealing its genetic makeup and potential applications in treating the bacterium. The newly released genome contains 2,326 sequenced genes, providing a comprehensive model for researchers to study its virulence and develop new therapies.
Researchers tested four viruses to transport genetic material into defective eyes of 18-day-old rats. Adenovirus proved the most effective vector, targeting specific tissues and preventing disordered blood vessel growth. Early-stage research suggests a potential breakthrough in treating retinopathy of prematurity with gene therapy.
Jefferson researchers successfully introduced 90 billion copies of a healthy gene into the brains of three children with Canavan disease. The goal is to increase enzyme production and reduce toxic compound buildup, potentially improving brain development and function.
Researchers warn that angiogenesis therapy could trigger growth in abnormal tissue, increase artery-clogging plaque, and stimulate inflammatory response. Despite these potential complications, the authors remain hopeful that the therapy will ultimately prove effective and safe.
Dr. Boucher is a leading researcher on cystic fibrosis, publishing over 300 articles and developing novel treatments. The University of North Carolina's genomics initiative receives $245 million in funding to advance research and therapies.
Dr. Jeffrey S. Chamberlain joins the University of Washington to study muscular dystrophy gene therapies with a focus on developing vectors for genetic delivery. His research aims to prevent and reverse the disease, offering hope for treatment or cure options.
Scientists at UNC Health Care have successfully used gene-therapy to produce high amounts of a clotting protein in animals, bypassing a major hurdle for treating hemophilia. The new method could potentially yield sustained factor IX production in humans, eliminating the need for daily injections.
Researchers developed a tissue culture system that mimics normal cell growth, allowing them to study gene transfer techniques in greater detail. They found that cancer cells can be easily infected while normal cells are poorly infected, suggesting a potential target for gene therapy.
Researchers at Jefferson Medical College successfully introduced a therapeutic gene into the brain of two children with Canavan disease, showing decreased NAA concentration and increased myelin. The gene transfer demonstrated positive neurological improvements in the patients, offering hope for future use of gene therapy for brain dise...