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 lung cancer assay measures gene activity in tumor tissue and provides a risk score to predict patient survival after surgery. The assay has been validated in two independent trials and shows promise in identifying patients at highest risk of mortality.
Gene therapy is poised to disrupt traditional treatment methods with its effectiveness in treating diseases such as hemophilia B. The technology has demonstrated feasibility and is expected to bring significant changes to the healthcare marketplace.
Researchers at Penn Vet & Scheie Eye Inst. successfully treated dogs with X-linked Retinitis Pigmentosa using gene therapy, correcting defects in the RPGR gene and restoring vision. The treatment targets both rod and cone cells, showing promise for treating other photoreceptor degenerations.
Researchers develop gene therapy to correct X-linked retinitis pigmentosa, a genetic defect causing peripheral and night vision loss. The technique replaces a malfunctioning gene with a normal one, supplying a protein for light-sensitive cells to function.
Researchers at the Gladstone Institutes have identified a key genetic mechanism linked to congenital heart disease, revealing the importance of epigenetics in fetal heart development. The study highlights the role of Ezh2 and Six1 genes in regulating healthy heart development, which can have profound health consequences later in life.
A University of Illinois study found that personalized prognostic tools and gene-based therapies can improve the survival and quality of life of glioblastoma patients. The researchers discovered new general and clinical-dependent gene profiles that can be used to predict patient outcomes and select targeted therapies.
Scientists have solved the three-dimensional structure of a newly discovered type of gene-targeting protein called TAL effector, which has a unique LEGO-like modular architecture. This discovery enables researchers to engineer the protein for targeted gene modification, genetic engineering, and corrective gene therapy.
Researchers distinguish cancer subtypes and provide a more complete understanding of Type 2 papillary renal cell carcinoma (PRCC2), an aggressive type of kidney cancer. They also identify genes involved in clear cell renal cell carcinoma (CCRCC) development and progression, highlighting PLK1 as a promising potential therapeutic target.
New research reveals that many bacteria try to fend off fluoride by throwing it out, and that the presence of this transport system indicates fluoride has antimicrobial properties. The discovery also highlights a genetic switch called riboswitches, which can be used to enhance fluoride's effects against bacteria.
Researchers used AAV vectors to increase NGF levels in the hearts of diabetic mice, preventing cardiac dysfunction and preserving blood flow. The study suggests NGF gene therapy may have tremendous therapeutic potential for treating diabetic cardiomyopathy.
A recent clinical trial in patients with hemophilia B showed that Factor IX gene therapy was able to convert severe hemophilia to moderate or mild disease. The treatment demonstrated a sustained therapeutic effect and eliminated or substantially reduced the need for standard protein replacement.
Researchers at St. Jude Children's Research Hospital and University College London have achieved early success with a gene therapy developed to treat hemophilia B. The treatment increased Factor IX levels in adults with the disorder, reducing the need for clotting factor injections to prevent bleeding episodes.
A Phase III clinical trial found that adding gemtuzumab ozogamicin to standard chemotherapy improved event-free and overall survival in newly-diagnosed acute myeloid leukemia patients aged 50-70. The treatment also reduced toxicity while providing therapeutic benefits.
Researchers at the Salk Institute have developed a new gene editing technique that uses patients' own cells to correct genetic mutations in the HBB gene, which causes sickle cell disease. The method repairs the beta-globin gene without introducing harmful genes into cells and appears to be more efficient than traditional techniques.
A new study finds that sewage treatment plants can be a significant source of antibiotic-resistant genes in waterways. Researchers detected three genes that make bacteria resistant to tetracycline antibiotics at a facility on Lake Superior, highlighting the potential for even high-tech plants to contribute to the problem.
Scientists have developed a new approach to gene therapy using site-specific recombinases from yeast and phages, allowing for precise genetic modifications. This technique has the potential to improve efficiency and effectiveness of experimental gene therapies while reducing side effects.
Researchers have successfully delivered replacement genes to patients with muscular dystrophy using a 'chimeric' virus. The study demonstrates the potential of customized gene therapy as a treatment option for this devastating disease.
Researchers have discovered a new way to build muscle by suppressing a natural inhibitor, resulting in mice and worms with super-strong muscles. This breakthrough could lead to treatments for age-related or genetics-related muscle degeneration, as well as applications for athletes and individuals with genetic muscular dystrophy.
Despite advancements in genetic profiling, breast cancer treatment remains non-personalized due to limited robust prognostic and predictive factors. Standard gene expression profiling has shown limitations in predicting chemotherapy benefit and prognosis for certain disease subsets.
Researchers are investigating a novel gene therapy approach using LG631 to improve tolerance and effectiveness of chemotherapy for glioblastoma, a devastating brain cancer. The study aims to prevent damage to bone marrow, enabling patients to receive higher doses with fewer side effects.
Researchers create super-strong mice and nematodes by reducing natural inhibitor function, leading to denser muscle fibers and increased energy delivery. This breakthrough could lead to treatments for age-related or genetically caused muscle degeneration.
A novel point-of-care genetic test has been shown to be clinically feasible and accurate, enabling rapid personalization of anti-platelet therapy for patients at risk. The study demonstrates that tailored treatment successfully protected all patients with the at-risk genetic variant from adverse events.
Researchers at Ohio State University Medical Center have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited form of blindness. The trial aims to treat choroideremia, a disease affecting 100,000 worldwide, and holds promise for other genetic causes of blindness like retinitis pigmentosa.
Researchers have identified a new potential cancer drug targeting EPHA7, a tumor suppressor protein associated with follicular lymphoma. The discovery demonstrates the power of functional genomics in translating genetic insights into therapies.
Researchers have developed a new animal model to study Progeria and tested a gene therapy that significantly extended the lifespan of mice. The treatment, using 'vivo-morpholino' antisense oligonucleotide technology, reduced progerin production and improved various parameters related to the disease.
A Scripps Research scientist has been awarded a $500,000 grant to study the genetic mutations associated with Parkinson's disease. The researcher will investigate two genes, LRRK2 and SGK1, which have shown a link between their mutations and reduced risk of Parkinson's disease.
Researchers will combine neuroscience and engineering to repair spinal injuries and hearing loss using gene therapy, flexible electronics, optical imaging, and human-machine interfaces. The partnership aims to bring new treatments to the clinic and alleviate human suffering caused by neurological disabilities.
Researchers at Wayne State University developed a novel gene therapy approach to restore vision in people with age-related macular degeneration and retinitis pigmentosa. A license agreement has been signed with RetroSense Therapeutics, paving the way for Phase I clinical trials.
Scientists at Ohio State University have created a technique called nanochannel electroporation (NEP) that allows for precise injection of genes and proteins into individual cells. The method uses electrical pulses to deliver therapeutic agents, with potential applications in cancer diagnosis and treatment.
Researchers successfully used induced pluripotent stem (iPS) cells to treat a mouse model of a rare genetic liver disease. The study demonstrates the potential for iPS cells to be used in human gene therapy to counter pathological effects and promote liver regeneration.
A new study by Loyola University Chicago Stritch School of Medicine could lead to improved gene therapies for conditions such as heart disease and cancer. Researchers found that a virus used in vaccines can also be tailored to cause less of an immune response in gene therapy applications.
Researchers developed a novel bipartite gene therapy approach to temporarily preserve photoreceptors in a mouse model of retinitis pigmentosa. The treatment targets defective phosphodiesterase metabolism, reducing cGMP and Ca2+ levels, and showing promise for treating this genetic disorder.
A genetic variant in the GLCCI1 gene may explain why some people with asthma do not respond well to inhaled corticosteroids. Researchers found that individuals with two copies of the variant responded only one-third as well to steroid inhalers as those with regular genes.
A study on mice shows that reducing IRS2 signaling can slow the progression of Huntington disease. Meanwhile, research also finds that circulating glucose levels can modulate neural control of desire for high-calorie foods in humans. These findings provide potential therapeutic targets and new insights into the biology of obesity.
Researchers identified 16 new gene regions associated with blood pressure and confirmed 12 previously discovered ones, providing insights into the genetic mechanisms of hypertension. The study's findings could lead to new therapeutic drug development and improved treatment options for individuals with high blood pressure.
Scientists developed an electrically neutral matrix to hold DNA fragments, which spontaneously align and form ordered structures at high concentrations. This technology has potential applications beyond gene therapy, including the delivery of chemical drugs.
Researchers at Mount Sinai School of Medicine have identified SUMO1 as a potential therapeutic target for treating and preventing the progression of heart failure. Gene therapy with SUMO1 improved cardiac function in human and pig hearts, suggesting its critical role in pathogenesis.
A novel gene therapy approach combining with radiation therapy has been found to be safe and effective in treating glioblastoma multiforme, a deadly form of brain cancer. The treatment stimulates an immune response against the tumor, producing an 'immunogene therapy' effect.
Researchers at UNC School of Medicine have devised a gene therapy cocktail that can treat some inherited diseases caused by misfolded proteins. The approach uses an adeno-associated virus (AAV) vector to deliver two payloads simultaneously: one disables the mutant protein and another provides a new gene to replace its activity.
Researchers at National Physical Laboratory develop a model peptide sequence dubbed GeT that transports genetic material into human cells, overcoming poor cell membrane permeability. This approach holds promise for gene therapy in treating defective genes such as cancer.
A new study develops and tests genetically engineered spider silk for safe and efficient gene delivery, offering a promising alternative to viral vectors. The material successfully attaches to diseased cells and injects DNA without harming mice in lab studies.
Researchers found that individuals lacking TLR1 proteins are more susceptible to tuberculosis, leading to decreased immune function. The study suggests a potential approach for personalized therapy and prioritizing drug treatment in developing countries.
A study published in The Lancet reveals that targeted antisense therapy AVI-4658 can restore expression of a key protein lacking in patients with Duchenne muscular dystrophy. The treatment showed significant dose response, allowing boys to produce functional dystrophin protein up to 18% of normal levels.
Researchers at Thomas Jefferson University have demonstrated the long-term therapeutic effectiveness and safety of S100A1 gene therapy in a large animal model of heart failure. The therapy works by restoring normal levels of the calcium-sensing protein, preventing and reversing cardiac dysfunction.
Researchers used gene transfer to deliver a therapeutic gene that produces protein apoA-1, which removes harmful cholesterol from atherosclerotic lesions. This approach protected against atherosclerosis in rabbit studies, with minimal and stable disease measurements over 48 weeks after a single dose of gene therapy.
A team of researchers led by UCSF and the Vaccine and Gene Therapy Institute aim to eradicate HIV from the body using a combination regimen. They will define HIV's reservoir, understand its creation and maintenance, and test potential treatments.
Researchers have developed a novel gene therapy system using nanoparticles that can be freeze-dried and stored for up to three months. The technology shows promise in treating brain cancer with high efficacy and minimal risk of complications, offering a potential alternative to traditional therapies.
Scientists at Fox Chase Cancer Center have identified a novel approach to develop targeted cancer therapies by reversing silenced genes through demethylation. The study suggests that specific proteins, such as thymine DNA glycosylase, can actively remove methyl groups from DNA.
Gene therapy using stem cells holds great potential for treating a range of diseases, thanks to advances in gene marking techniques, PCR sequencing, and chromatin insulators. The success of these methods could lead to improved patient survival rates.
A new study found that a specific gene variant, PNPLA3 rs738409, is linked to an increased risk of developing fatty liver and faster fibrosis progression in people with chronic hepatitis C. The study suggests this genetic predictor may also be used as a potential therapeutic target for treating the condition.
A team of researchers has discovered a naturally occurring disease in monkeys that shares similarities with multiple sclerosis, providing new insights into the cause of the disease. The discovery is associated with a herpes virus and could lead to significant advancements in MS research and treatment.
Researchers at Mount Sinai School of Medicine developed a gene therapy called SERCA2a that stabilized or improved cardiac function in people with severe heart failure. Patients receiving the high-dose therapy experienced substantial clinical benefit and significantly reduced cardiovascular hospitalizations.
New tools and methods enable targeted gene delivery to cells in the central nervous system, offering hope for new treatments. Novel strategies include using stem cells and siRNAs to silence genes involved in neurological diseases.
Researchers used genome editing to treat hemophilia in mice by precisely targeting and repairing mutated DNA. The treatment, which uses zinc finger nucleases, showed clinically meaningful results with no toxic effects or complications.
Researchers at the University of Pennsylvania have developed a new gene vector, AAV8, that delivers genes to photoreceptor cells more efficiently and safely than a previously used vector, AAV2. The study demonstrates potential for treating eye diseases such as retinitis pigmentosa.
Research suggests that many hereditary conditions are linked to errors in pre-mRNA splicing, which can be addressed through new therapies. A computer analysis predicts that 22 percent of genetic mutations may affect splicing, implying hundreds of hereditary diseases could be linked to this issue.
An experimental gene therapy has reversed type 1 diabetes in mice with a nearly 80 percent success rate, reversing autoimmune destruction of insulin-producing beta cells. The treatment uses neurogenin3 and betacellulin to stimulate new islet growth and inhibits immune system activity.
Research at the University of Texas MD Anderson Cancer Center found that customizing targeted therapies based on a patient's specific gene mutations can lead to higher response rates, survival, and failure-free survival compared to non-matched patients. This approach has shown promise in treating solid tumors with gene aberrations.
A gene discovery for migraine may lead to the development of a preventive pill that blocks the brain's pain channel. The discovery is significant as it opens understanding of the pain pathways that trigger migraine pain, sparking excitement among researchers and the pharmaceutical industry.
Researchers found loss of PTEN and mutations in MEK2, CDK4, and CDKN2A to be associated with patient response to GSK436. This study provides new insights into personalized approaches to melanoma therapy.