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 study found that a specific gene change can predict which brain cancer patients respond well to treatment, allowing for personalized therapy. The gene MGMT methylation status was associated with improved overall and progression-free survival in patients treated with radiation and chemotherapy.
A phase 3 trial of a novel gene therapy failed to reduce amputations or deaths in patients with critical limb ischemia. The study highlights the need for effective treatments for this condition, which affects millions worldwide and results in significant healthcare costs.
Neurologix presents 1-year data from its Phase 2 study of NLX-P101, a gene therapy for Parkinson's disease, showing sustained improvements in motor symptoms. Patients treated with NLX-P101 experienced highly clinically meaningful symptom improvements, with a mean 37% improvement after one year.
A National Institutes of Health grant has been awarded to Nationwide Children's Hospital to develop a gene therapy for MPS IIIB, a fatal lysosomal storage disease. The therapy aims to deliver needed genes directly to the central nervous system by bypassing the blood-brain barrier.
Researchers have successfully isolated individual charged particles like DNA molecules using a method called Paul trapping, which could lead to personalized diagnosis and treatment. The device is small, inexpensive, and can be used on a single chip, allowing for rapid testing of patient samples in offices.
Researchers identified a new probiotic bacteria-derived soluble protein that can protect intestinal cells from inflammation and injury in mice models of colitis. The protein's delivery to the colon provided therapeutic protection against ulcerative intestinal inflammatory disorders.
Researchers at Salk Institute successfully edit a diseased gene in patient-specific induced pluripotent stem cells and adult stem cells using a virus-based approach. The method provides an efficient and safe tool for cell engineering, opening the way for gene editing-based stem cell therapies suitable for clinical applications.
Advances in gene therapy success depend on the development of viral delivery vectors. Researchers have made progress in refining AAV-based vectors, improving their production and use. This has enabled clinical proof of concept and paved the way for commercialization.
Researchers combine Sabutoclax with viral gene therapy to prevent tumor growth in prostate-cancer-prone mice, offering a novel approach to treating advanced prostate cancer. The study's findings suggest that this combination therapy could be effective in other cancers and pave the way for personalized medicine.
Recent research highlights exciting developments in ocular gene therapy, including potential treatments for age-related macular degeneration and retinal disease. Gene therapy strategies have shown promise in reducing cell death and slowing retinal degeneration.
Researchers at Tufts University School of Medicine developed a gene therapy approach that significantly reduces uncontrolled blood vessel growth and cell death typical of AMD, slowing its progression. The treatment uses CD59 to block the formation of 'membrane attack complex' in cell membranes.
Researchers developed an effective gene therapy technique using induced pluripotent stem cells to correct chronic granulomatous disease in cell culture. The corrected neutrophils produced normal levels of hydrogen peroxide, potentially offering a cure for the rare disorder.
Researchers at University of Michigan have developed a novel gene therapy, NP2, that shows promise in providing substantial pain relief for patients with unrelenting cancer-induced pain. The study demonstrates the safety and efficacy of NP2 in reducing pain by up to 80% over four weeks.
Researchers have determined that correcting a genetic defect does not substantially increase the number of potentially cancer-causing mutations in induced pluripotent stem cells. This breakthrough suggests that human-induced pluripotent stem cells altered to correct a genetic defect may be cultured into subsequent generations of cells ...
A study found that young adult patients with hypertrophic cardiomyopathy survived cardiac arrest after receiving therapeutic hypothermia. The treatment was successful despite marked left ventricular wall thickness and other risk factors.
Researchers successfully reprogrammed diseased human hepatocytes into induced pluripotent stem cells, offering a potentially unlimited source for liver disease treatment. This breakthrough could enable the generation of genetically corrected liver cells via auto-transplantation, avoiding liver transplants and immunosuppression.
A double-blind gene therapy trial has demonstrated safety and significant improvement in motor function for patients with Parkinson's disease. The study found that injecting the glutamic acid decarboxylase (GAD) gene directly into the brain improved motor scores by 23.1% compared to a placebo group.
A gene therapy called NLX-P101 significantly reduces movement impairment in Parkinson's patients, with half achieving dramatic symptom improvements. The treatment introduces a gene to normalize chemical signaling, correcting dysfunction in brain circuitry responsible for coordinating movement.
A new study found that gene therapy NLX-P101 improved motor performance and control of Parkinsonism symptoms in half of patients, offering hope for the treatment of neurodegenerative disorders. The therapy has been safe and well-tolerated over a six-month period.
A double-blind trial demonstrated a 23% improvement in motor symptoms among patients receiving gene therapy for advanced Parkinson's disease, compared to a 12% improvement in those receiving sham surgery. The study suggests a potential breakthrough in treating this progressive movement disorder.
A study at Cold Spring Harbor Laboratory has identified a therapeutic target for liver cancer using a monoclonal antibody that blocks the activity of the FGF19 gene. This approach shows promise in treating patients with FGF19 amplification, which can be used as a biomarker to predict treatment effectiveness.
Researchers have identified 15 new genetic regions associated with an increased risk of developing primary biliary cirrhosis. The study used genome data from 2,500 patients and 7,500 healthy individuals, tripling the number of known genetic regions to 22.
Researchers believe ADAM-12 gene could be important in fighting cancer, arthritis and cardiac hypertrophy. The gene's regulation pathway could lead to new treatment plans.
A new patented therapy has been developed by WSU researcher Dr. Karli Rosner, which uses genetic constructs to target cancer cells and induce apoptosis, eliminating the need for external medications. The treatment shows promise in treating various types of cancers, including melanoma, prostate, lung, and breast cancers.
Researchers identified AEG-1 as a strong predictor of progression-free survival in patients with EGFR-mutant non-small cell lung cancer treated with erlotinib. Patients with low AEG-1 expression had longer progression-free survival compared to those with high expression.
Researchers discovered a rare gene mutation strongly linked to schizophrenia, which could be a new target for effective treatment. The mutation affects the VIPR2 gene, leading to increased signaling in the Vasoactive Intestinal Peptide pathway.
A team of investigators has identified a new candidate gene for primary open angle glaucoma (POAG), the most common form of eye disorder. The gene, ADAMTS10, is involved in processing extracellular matrix and may play a role in regulating aqueous humor outflow.
A new gene test has identified GFPT1 as a crucial cause of Congenital Myasthenic Syndrome (CMS), a rare genetic condition affecting signal transmission between the brain and muscles. The test allows for earlier treatment with cholinesterase inhibitors, offering an effective therapy that can be taken through life.
Researchers have developed a new method for treating genetic diseases using modified mRNAs, which can be administered repeatedly without increasing the risk of cancer or severe immune reactions. In mouse models, this technique successfully restored lung function in mice with a congenital lung defect.
Researchers develop an innovative genetic strategy that transfers a bacterial gene into CD4+ T-cells, making them resistant to HIV infection. The MazF protein destroys gene transcripts, preventing protein synthesis and effectively blocking HIV replication.
Researchers found that adding caffeine to lentiviral production protocols increases virus production by 3-8 times. This simple and inexpensive strategy may significantly decrease the cost of lentiviral production for research and clinical uses.
Two studies found that ITPA variants protect against treatment-related anemia in HCV patients, enabling safer use of antiviral therapy and reducing the need for erythropoietin treatment. The variants also help select patients who may tolerate higher doses of ribavirin, increasing the likelihood of viral clearance.
Researchers at Duke University Medical Center discovered how nanoparticles from diesel exhaust damage lung airway cells, a finding that could lead to new treatments for people susceptible to airway disease. The study showed that the severity of injury depends on an individual's genetic make-up.
Dr. Sena-Esteves is investigating gene therapy techniques to replace the faulty gene causing Tay-Sachs Disease, a devastating neurological disorder. He's part of the Tay-Sachs Gene Therapy Consortium aiming to translate current results into a human clinical trial within three years.
Researchers have created a new type of nanoparticle called nanodisks, which can carry genetic material and enter cells quickly without losing its potency. These nanodisks offer a promising alternative to traditional gene therapy vectors, potentially improving therapeutic outcomes.
Researchers from the University of Leicester and international partners have identified potential new therapeutic targets for Huntington's disease using cutting-edge genetic techniques. The study found that flavonoids can prevent cell death in yeast, suggesting a possible new treatment approach.
Researchers developed a gene therapy strategy using parthenogenetic embryonic stem cells, which can differentiate into multiple tissue types and potentially reduce rejection problems. The approach shows promise for treating autosomal dominant diseases like Beta Thalassemia, tuberous sclerosis, or Huntington's disease
A recent study published in the New England Journal of Medicine found that losing the NFKBIA gene promotes glioblastoma multiforme growth and reduces survival. Restoring this gene may improve survival for certain patients with glioblastoma, as it inhibits tumor cell growth and increases sensitivity to chemotherapy.
A new, simplified method for producing large amounts of viral vector cassettes has been developed, overcoming previous limitations. This approach enables efficient packaging of single- or double-stranded AAV vectors and large AAV cassettes, paving the way for commercial production.
Researchers have developed a novel virus-based gene therapy for renal cell carcinoma, killing cancer cells both locally and distantly. The therapy combines Sorafenib with Ad.5/3-mda-7 adenovirus, which activates the MDA-7/IL-24 protein to stop tumor growth.
Scientists at Johns Hopkins and VCU have developed a nanoparticle-based gene 'ignition switch' to detect and treat cancer. The promoter-based system activates specific genes only in cancer cells, showing a 30-fold difference in identification.
A new $2.1 million NIH grant will help MU scientist Dongsheng Duan develop a treatment that prevents heart muscles from weakening due to Duchenne muscular dystrophy. Tests have shown that treating skeletal muscle alone can lead to complications, highlighting the need for comprehensive treatment targeting both skeletal and cardiac muscle.
Researchers at the University of Cincinnati found a potential genetic target, miR765, which can down-regulate protein phosphatase 1 inhibitor-1 and reduce cardiac muscle contractility. This micro-RNA may play a role in heart disease development by decreasing or deactivating proteins that help the heart function at full capacity.
A new study found significant changes in targeted genes in patients' tumors, suggesting existing therapies may benefit more cancer patients. The research identified potential targets for treatment with existing targeted therapies.
Researchers achieved marked clinical improvements in two young children with Wiskott-Aldrich syndrome through gene therapy, correcting immunodeficiency and reducing complications. The treatment is a promising step forward for patients with this rare and severe disorder.
Researchers have developed a faster-growing E. coli strain suitable for mass production of high-quality DNA for vaccines and gene therapy. The modified strain retains useful traits while growing as quickly as other industrial strains.
Dr Ross Dickins, a leading cancer researcher at Walter and Eliza Hall Institute, has been awarded a $975,000 fellowship from the Sylvia and Charles Viertel Charitable Foundation. The fellowship will support his work on identifying the normal function of genes recently implicated in leukemia.
A recent review reveals that antibiotics can alter normal gut bacteria composition and leave resistance genes present for extended periods. This increases the risk of pathogenic bacteria survival and multi-drug resistance.
Antigene therapy utilizes a DNA-based drug that attaches directly to specific DNA sequences and is activated by light energy, effectively silencing targeted genes. This approach may offer new hope for treating diseases currently incurable or having limited success.
Recent advances in neurogenetics research have shed light on the genetic basis of human brain disorders, including Parkinson's and Alzheimer's diseases. Genetic discoveries have transformed clinical practice and provided new hope for patients and families affected by these devastating disorders.
Researchers at NewYork-Presbyterian/Weill Cornell report that gene therapy to the brain reverses depression-like behavior in mice, with human data backing up the promise of such therapy. Autopsy studies reveal significantly reduced levels of p11 protein in patients with severe depression.
Scientists at the University of Michigan have identified four new DNA hotspots that may aid in developing new treatments for psoriasis. The study, published in Nature Genetics, builds on past work by U-M researchers and provides a better understanding of the hereditary factors of the disease.
Researchers studied genetic risk factors for age-related macular degeneration (AMD) and found that patients carrying specific genes were more likely to respond to anti-VEGF therapy. Genetic screening may improve glaucoma care by identifying markers for congenital and primary open angle glaucoma.
Cincinnati Children's Hospital Medical Center has been named a national Center of Excellence for Molecular Hematology to accelerate the discovery of new therapeutic approaches for conditions like sickle cell anemia and leukemia. The center will also help speed the transition of new therapies from research to clinical trials.
A clinical trial of gene therapy for muscular dystrophy has uncovered an unexpected aspect of the disease, revealing that some patients mount an immune response to the dystrophin protein even before receiving treatment. The study suggests that tailoring treatment approaches to suit individual patient needs could lead to better outcomes.
Researchers studying gene therapy for Duchenne muscular dystrophy discovered a natural immunity to dystrophin, complicating experimental therapies. The immune response triggered by T cells may target muscle cells, resembling autoimmunity.
A new, fully characterized viral vector is introduced as a reference material to standardize gene therapy protocols in research applications and human clinical trials. The availability of this reference standard allows vector parameters to be expressed in common units, enabling comparison and normalization across laboratories.
The NIH Director's Transformative Research Projects (T-R01) award program provides funding for exceptionally innovative research ideas that have the potential for extraordinary impact. The program allows investigators to sidestep conventional stumbling blocks and propose daring ideas.
A new study has found prenatal cardiac defects in mice with Spinal Muscular Atrophy (SMA), a leading genetic cause of infantile death. The discovery has significant implications for treatment, suggesting that the disease may be multisystemic and requiring therapies beyond just motor neurons.
Research at OHSU's Vaccine and Gene Therapy Institute suggests that G-CSF, a hormone stimulating stem cell growth, can reactivate human cytomegalovirus in bone marrow recipients, increasing their risk of infection. The study highlights the need for factoring in the risks associated with G-CSF use during transplants.