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 novel method using immune cells has been shown to induce tolerance to specific proteins in mice, allowing them to tolerate gene therapy designed to deliver the protein. This approach may prevent rejection and improve the long-term success of gene therapies for various diseases.
Researchers at the University of Cincinnati have identified a new genetic target, pendrin, for diuretic therapy. The study suggests that targeting pendrin could lead to a targeted treatment option for patients with severe fluid overload who may not respond well to current diuretics.
A team of international scientists has identified the new gene responsible for Leber Congenital Amaurosis (LCA), a devastating genetic form of blindness in newborns. NMNAT1 is crucial for life and produces a vital coenzyme called NAD, involved in hundreds of reactions in the cell.
A recent study published in Cancer Biotherapy and Radiopharmaceuticals suggests that high-dose interleukin-2 (IL-2) should continue to be the initial treatment for patients with stage IV metastatic melanoma. The researchers recommend intensive IL-2 therapy as a viable option, either alone or in combination with newer therapeutic agents.
A new gene therapy approach successfully treats genetic hearing loss in mice by delivering the VGLUT3 gene to inner ear cells. This breakthrough offers a promising potential treatment for individuals born deaf, with improved hearing lasting up to 1.5 years in adult mice.
A novel gene therapy approach has been developed to increase frataxin protein levels in Friedreich's ataxia patients. The method, using TALE proteins, successfully boosted frataxin production by 2-3 fold, offering a potential solution for treating the genetic disorder.
Researchers at the University of Missouri have developed a gene therapy treatment that extends the lives of mice with spinal muscular atrophy by introducing a missing gene into their central nervous systems. This breakthrough offers new hope for treating humans with SMA, potentially providing a cure within 12-18 months.
A new gene therapy approach has been developed to deliver full-length versions of large genes, improving skeletal muscle function in patients with genetic disorders like dysferlinopathies. The strategy may hold new hope for treating limb girdle muscular dystrophy type 2B and other muscular dystrophies.
A novel gene therapy strategy using hyperbranched poly(amidoamine) nanoparticles has been developed for cardiac repair. The strategy, known as HRE-VEGF, provides a safer alternative to current VEGF gene delivery systems and shows promise for treating myocardial infarction.
Researchers at the University of Cincinnati discovered a new gene target for leukemia therapy by inhibiting the protein Vav3, which controls cell signaling. This finding could lead to cellular targets for patients with BCR-ABL lymphoid leukemia, a type of leukemia with poor treatment outcomes.
Researchers have identified eight new genetic variants associated with osteoarthritis, which could lead to future therapeutic intervention. The study suggests that inherited factors account for up to 60% of the risk, and the discovery provides insight into the genetics of arthritis.
Scientists at Scripps Research Institute discovered a simple and safe method to disrupt specific genes within cells, offering a potential HIV treatment. The new technique uses zinc finger nuclease proteins, which can be added directly to cells without viral delivery methods.
Gene therapy successfully replaces protein missing in Pompe disease when targeting liver cells, reducing immune system reaction. Combining liver-expressing vector with ubiquitously expressing vector boosts overall effectiveness of the treatment.
Researchers at University of North Carolina Health Care have discovered a new role for the SMN gene in spinal muscular atrophy, contradicting previous assumptions. The study suggests that faulty processing of genetic material is not the primary cause of the disease, but rather a separate function of the SMN gene.
Gold nanoparticles with a slight positive charge have been found to unravel DNA's double helix, enabling potential breakthroughs in gene therapy. The research also highlights the importance of characterizing nanoparticle characteristics for DNA-based electronics.
Researchers developed a single-dose vaccine that produces an antibody capable of binding to cocaine, preventing its entry into the brain. The vaccinated mice showed no effects from intravenous cocaine exposure, highlighting a potential novel approach to treating cocaine addiction.
Researchers have developed a device that delivers a genetically engineered neurotrophic factor directly to the brain, treating neurological symptoms in laboratory rats. The study found significant protection against toxicity and reduced striatal lesion size, suggesting promising therapeutic potential for Huntington's disease.
Researchers at UMass Chan Medical School have identified the first gene, dSarm/Sarm1, responsible for promoting axon destruction after injury. The discovery provides a new therapeutic target to delay or stop axon decay in neurodegenerative diseases.
Researchers at Children's Hospital of Philadelphia have developed approaches to control long-range genomic interactions during gene expression. By identifying a looping factor, they showed that chromatin looping is a cause, not an effect, of gene transcription.
Researchers at Griffith University are on the brink of an effective human treatment for the deadly Hendra virus, which has killed over 200 people in South East Asia. The therapy attacks the virus by turning off a vital gene and is highly effective in very low doses.
A new study suggests that genetic variations can predict a person's response to drug treatments for nicotine addiction, with those at high-risk of heavy smoking showing the greatest response to pharmacologic therapy.
Researchers at Ohio State University Comprehensive Cancer Center discovered a small inherited change in DNA responsible for overactivating the BAALC gene, which is associated with poor treatment response in acute leukemia. The study found that high levels of RUNX1 protein are linked to high BAALC gene expression.
A recent clinical trial found that gene therapy can insert the correct ADA gene into patient bone marrow cells, leading to a good response. Defects in B cell tolerance are also corrected after gene therapy, supporting its use as an effective treatment option for ADA-deficient severe combined immunodeficiency patients.
Researchers developed a new method to identify vaccine targets for Streptococcus pneumoniae, the most common cause of bacterial meningitis. Gene therapy has shown promise in treating adenosine deaminase deficiency by correcting B cell tolerance problems and supporting treatment options for patients with severe combined immunodeficiency.
Experimental video game technology helps therapists monitor patients' arm rehabilitation after stroke, improving recovery outcomes. A UK-led gene therapy approach has shown promising results in treating hereditary blindness, with no adverse effects reported.
Taiwanese doctors use gene transfer to treat children with a rare neurological disease, restoring movement and improving cognitive development. The treatment has implications for Parkinson's disease and other neurodegenerative disorders.
Researchers found that injecting DNA nanoparticles increased expression of indoleamine 2,3 dioxygenase (IDO), an enzyme that calms the immune response, significantly reducing limb joint swelling and inflammation in rheumatoid arthritis. This suggests a potential therapeutic value for gene therapy in autoimmune diseases.
Researchers found two specific gene variants associated with faster motor decline in Parkinson's patients. These variants can now help doctors predict disease progression, allowing for targeted therapies and faster results on efficacy of drugs.
Researchers at CNIO successfully test first gene therapy to combat aging, extending mouse lifespan up to 24 percent and improving health. The therapy delivers a 'rejuvenating' effect using telomerase enzyme, repairing or delaying DNA damage.
A UNC-led team discovers that lung cancer molecular subtypes correlate with distinct genetic alterations and patient response to therapy. The study refines their previous report on three molecular subtypes of non-small cell lung cancer, suggesting new avenues for targeted treatment.
Scientists at NC State University developed a method to turn gene expression on and off using light-activated molecules. This technique enables precise control over gene function, potentially leading to targeted therapies for diseases like cancer.
Positive results from animal models and initial clinical trial results show promise for retinal gene therapy to treat inherited diseases. Researchers have developed efficient and safe viral delivery systems to introduce therapeutic genes into photoreceptor cells.
Researchers from the University of Pennsylvania report that genetically modified T cells remain healthy up to 11 years after initial therapy in a decade-long study of HIV patients. The approach provides a framework for gene therapy as a powerful weapon in treating HIV, cancer, and other diseases.
A geneticist has developed a technique to test the functional differences of human gene variants using yeast cells. This method identified 37% of alleles associated with vitamin B6 supplementation for treating homocystinuria, a metabolic disease causing blood clots and mental retardation.
Researchers at Bascom Palmer Eye Institute have developed a novel gene therapy to treat Leber Hereditary Optic Neuropathy (LHON), an inherited genetic defect that causes rapid vision loss. The treatment successfully replaces mutated genes with healthy ones, restoring visual function in experimental models.
Researchers found vitamin D can stimulate bone cells to remove calcium from bones, potentially leading to density loss and increased fracture risk. This discovery may explain conflicting results of clinical trials examining the effects of vitamin D supplements on preventing fractures in elderly patients.
Scientists at IRB Barcelona and BSC successfully extracted structural information from a triple DNA helix in gas phase, preserving its biological environment. This breakthrough could lead to the development of antigen therapy based on these DNA structures.
Researchers use groundbreaking gene sequencing technology to rapidly detect FLT3 mutations in AML patients who have relapsed on therapy. This discovery may help develop new therapies to treat AML, a type of leukemia characterized by rapid white blood cell growth.
Scientists have developed a new technique to determine which patients with homocystinuria are most likely to respond to vitamin B6 treatment based on their genotypes. The study correlates specific gene mutations with disease severity and may help physicians prescribe treatment based on genotype.
A preclinical study found that patients with KRAS gene mutations have a worse prognosis and do not respond well to targeted therapies. However, patients with KRAS-mutant tumors may respond well to treatment with antifolates, especially if the mutation is not amplified.
Researchers have discovered gene variations that predispose cystic fibrosis patients to develop an intestinal blockage while still in the uterus. These variants involve genes responsible for ion transport in the lower end of the small intestine, increasing the risk of meconium ileus and serious health problems.
Cardiomyopathy is a deterioration of the heart muscle affecting its pumping ability. MDC-researchers identified RNA binding motif protein 20 (RBM20) as a gene regulating titin splicing, a process connected to the disease. Understanding this mechanism may lead to more efficient molecular diagnosis and therapies for cardiomyopathy.
The University of Ottawa Heart Institute's bedside genetic test has successfully protected patients with a specific genetic variant from adverse events. The test uses a simple cheek swab and has been shown to be effective in reducing high on-treatment platelet reactivity, a marker for complications after stenting.
Researchers have designed a powerful gene therapy strategy to treat beta-thalassemia and sickle cell anemia by transferring a healthy beta-globin gene into diseased cells. The new technique has shown promising results, with increased production of normal hemoglobin in patients.
Researchers found that certain genetic mutations in acute myeloid leukemia patients predicted improved outcomes when treated with high-dose induction chemotherapy. Mutational profiling could help identify distinct subgroups of patients who may benefit from dose-intensified therapy.
A genetic pathway previously known for its role in embryonic development and cancer has been identified as a target for systemic sclerosis, or scleroderma, therapy. The finding reveals that the Wnt signaling pathway is abnormally activated in scleroderma patients, leading to fibrosis and tissue damage.
A groundbreaking gene therapy trial for cystic fibrosis will begin in March, involving 130 adults and children with the disease. The trial aims to assess whether repeated doses of gene therapy can improve symptoms and lung function in patients.
Researchers have discovered a dramatic improvement in life span and motor function in mice with infantile Batten disease when treated with gene therapy and bone marrow transplants. The combination therapy created a striking synergy, with mice living nearly 18.5 months, more than double the lifespan of untreated mice.
Researchers found gene mutations in DNMT3A and NPM1, as well as translocations, associated with improved patient outcomes when treated with higher doses of daunorubicin chemotherapy. This discovery could help physicians tailor treatment plans for individual patients.
Researchers have identified genetic abnormalities that help doctors predict patient prognoses and guide treatment decisions for AML. The study shows that nearly two-thirds of patients can be categorized into clear prognostic groups, leading to improved treatment outcomes.
A genetic signature has been identified for eosinophilic esophagitis (EoE), a disease causing painful food allergies, inflammation, and swallowing difficulties. The microRNA signature is reversible with steroid treatment, offering an opportunity for non-invasive diagnosis and precise analysis.
Researchers successfully deliver genes for PDGF-BB and VEGF to mice, restoring blood flow and repairing damaged tissue without toxic effects. The balanced delivery of these factors shows promise for treating atherosclerotic disease and preventing limb amputations.
Researchers have found that an epigenetic blockade affects people with Alzheimer's disease, leading to a blockade of genes involved in learning and memory. A treatment approach using gene therapy reduced HDAC2 levels, preventing the blockade and improving neuroplasticity.
A study in a mouse model of Down syndrome identifies increased expression of protein Dyrk1a as a promoter of acute megakaryoblastic leukemia, offering a candidate therapeutic target for treatment.
A computer model used by researchers at Stanford University found that new triple-therapies for genotype-1 hepatitis C are cost-effective for patients with advanced disease. The treatment options have more severe side effects and boost costs, but help these patients avoid costly cancers and liver transplants.
Researchers report growing success in combining therapeutic cancer vaccines with conventional chemotherapy, stimulating the immune system to destroy cancer cells. The first FDA-approved cancer vaccine is for metastatic prostate cancer, and several other strategies are being tested for various types of cancer.
Researchers successfully improved vision in three adult patients who previously received gene therapy in one eye, achieving better light sensitivity and navigating obstacles in dim light. No immune reactions occurred during the readministering treatment, and unexpected benefits were observed in brain responses.
Researchers found a gene, LTA4H, that influences inflammation to TB infection, predicting drug therapy effectiveness. This discovery suggests tailoring treatment based on patients' genetic sequences could improve patient outcomes.
Researchers have discovered new mechanisms by which RNA drugs can control gene activity, including binding to Argonaute proteins and other forms of non-coding RNAs. This breakthrough provides exciting new options for targeting emerging forms of non-coding RNAs and pre-mRNAs.
Researchers found that a single 'Goldilocks' gene, LTA4H, regulates the immune response to TB. Variations in this gene lead to excessive or insufficient inflammation, which can affect treatment response. The study suggests personalized medicine could improve TB treatment outcomes.