Articles tagged with Gene Therapy
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.
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 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.
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.
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.
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 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.
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.
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.
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.
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.
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.
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.
Scientists at the University of Granada have developed a new therapy for skin and lung cancer using a suicide coliphage-gene, demonstrating effectiveness in vitro and in vivo. The treatment involves inducing cell death in tumour cells, potentially reducing the need for chemotherapy.
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.
Researchers found that inhibiting GRK2 protects heart cells from destruction after a heart attack, while excess GRK2 leads to more damage. The discovery suggests that anti-GRK2 gene therapy may help prevent permanent heart damage.
A Phase 1/2 study of LentiGlobin gene therapy has demonstrated positive results in a young adult with severe beta-thalassemia, achieving transfusion independence for over two years. The treatment also identified cells with the corrected beta-globin gene that overexpressed HMGA2.
Researchers are calling for a new focus on hypertrophic cardiomyopathy (HCM), a genetic disorder causing sudden death in young people. A task force of cardiologists and cardiac biologists recommends studying the natural history of HCM, defining all genetic causes, supporting clinical trials, and preventing mutant gene expression.
Scientists at Duke University Medical Center have identified a gene associated with myopia in Caucasian people, providing new hope for gene therapies. The RASGRF1 gene plays a crucial role in retinal function and visual memory consolidation, making it a promising target for treatment.
A new study reveals that CEP290 is crucial for maintaining the structural integrity of the ciliary gate, a key component of cilia. This discovery could lead to targeted gene therapy for cilia-related disorders, including Meckel syndrome and Joubert syndrome.
A multidisciplinary team at UCLA has successfully visualized a virus using cryo-electron microscopy, enabling the adaptation of the adenovirus for use in gene-therapy treatments for prostate and breast cancers. The breakthrough provides critical structural information for researchers attempting to modify the adenovirus.
A team of 64 international researchers has identified 27 new MiniPromoters, greatly expanding the current limited pool of these vital tools. The Pleiades Promoter Project enables research worldwide with bioinformatics, genomics, and neuroimaging expertise.
Researchers found a genetic rarity where disease-causing genes can self-repair, resulting in spots of normal skin amidst diseased skin. The recombination of chromosomes prior to cell division leads to cells with either two mutant copies or no mutant copies, forming normal skin patches.
Researchers at Scripps Research Institute have determined the atomic structure of a human adenovirus, leading to insights into its assembly and potential applications in gene therapy. The largest complex ever solved at atomic resolution, this discovery may lead to more effective treatments for diseases such as cystic fibrosis and cancer.
A recent study by UCLA researchers has identified a genetic variation linked to systemic lupus erythematosus (SLE) in Asian men. The findings suggest that this variation on the X chromosome enhances an immune response, increasing the risk of developing lupus in men compared to women.
Researchers at Tufts University develop nanoparticles to deliver therapeutic genes to the retina, delaying onset of eye disease and preserving vision. The treatment, using a gene for GDNF, shows temporary but significant protection against photoreceptor cell death.
Researchers successfully replaced a human gene to preserve photoreceptor function in mice with severe retinal degeneration. The study demonstrates improved function and survival of photoreceptors, validating a potential gene therapy design for patients affected by Leber congenital amaurosis.
Scientists have developed a new approach to deliver therapeutic genes into brain cancer cells, using chlorotoxin from deathstalker scorpion venom and nanoparticles of iron oxide. This method has shown promise in inducing nearly twice the amount of gene expression in brain cancer cells compared to traditional approaches.
Gene therapy holds promise in treating prostate cancer, but challenges remain in achieving effective treatment. Researchers call for better testing systems and innovative approaches to advance the field. Prostate cancer is a leading cause of cancer death among men in the US.
Italian scientists have successfully completed preclinical trials for a gene transfer treatment that can correct the lack of beta-globin in patients' blood cells, a major step towards treating beta-thalassemia. The treatment uses genetically corrected stem cells to restore haemoglobin production and overcome the disorder.
Researchers at Biogen Idec Inc. have found that targeting the TIM-1 protein may be effective in treating asthma. The study used a humanized mouse model to show that an antibody binding to a specific region of TIM-1 reduced inflammation and airway hyperresponsiveness.
A genetically reprogrammed herpes virus and an anti-vascular drug combination shrinks spreading distant sarcomas in mice, with significant tumor reduction and improved survival rates. Researchers achieved these results by using the virus systemically via injection, overcoming a major stumbling block in previous cancer treatments.
Researchers create nanoparticle that effectively delivers genetic material into cells, outperforming existing methods. The device, a vector, is between 2 ½ and 10 times more effective than other experimental materials, according to the research.
A personalized approach to smoking cessation is developing, combining genetic information and smoking habits to predict effective nicotine replacement therapy. This method could increase cessation success rates by up to 25%.
Researchers developed an experimental cure for Type 1 diabetes using gene therapy, which successfully reversed the disease in about half of nonobese diabetic mice. The treatment protected new beta cells from autoimmune attack by adding a protective gene, allowing them to function normally.
A study found that patients with PTSD who carried a short allele of the serotonin transporter gene promoter responded more poorly to cognitive behavior therapy. The researchers suggest that genetic variation may influence treatment response, leading to potential modifications in treatment approaches.
Researchers at the University of Montreal have identified the mechanism behind three cancer-causing genes in acute lymphoblastic leukemia. The study provides insight into the complex interaction between these genes and their contributions to leukemia, which could lead to the development of less invasive cancer therapies.
Research on prion disease found that structural differences affect transmission between species, while gene therapy showed promise in nonhuman primates. Additionally, the protein periostin was discovered to promote heart valve degeneration by inducing angiogenesis and MMP production.
A small genetic change in the IL28B gene can predict how well individuals with hepatitis C will respond to treatment. Those carrying this protective allele are more likely to clear the virus and respond to therapy.
Researchers at the University of Oklahoma Health Sciences Center have found a way to use gene therapy to treat retinitis pigmentosa, an eye disease that causes blindness. The treatment involves delivering genetic therapies directly to light-sensitive cells in the retina using nanoparticle technology.