Articles tagged with Gene Therapy
A new study found that delivering gene therapy to the cerebrospinal fluid directly corrects gene mutations causing CLN2 disease, showing potential therapeutic effects. In nonhuman primates, TPP-1 activity increased significantly after treatment, providing hope for families affected by this fatal childhood disorder.
Researchers at Rice University have developed a non-invasive gene delivery technique using ultrasound to efficiently deliver clinically used gene therapy vectors throughout the brain. The study, published in Gene Therapy, shows that opening more sites within targeted regions improves gene delivery efficiency.
Scientists at NeuroRestore have developed a gene therapy that stimulates nerve regrowth and guides nerves to reconnect to their natural targets, restoring mobility in mice with complete spinal cord injuries. The treatment, tested in mice, shows promise in reversing paralysis and improving motor function.
Researchers at St. Jude Children's Research Hospital refined the definition of hyperdiploidy in childhood B-cell acute lymphoblastic leukemia (B-ALL), using DNA index to predict patient outcomes. The study found that a simpler system captures a significant proportion of patients with excellent prognoses, and that individual chromosome ...
A team of UC Irvine scientists discovered a llama-derived antibody called a nanobody that can halt Rhodopsin's misfolding and activation, potentially treating Retinitis Pigmentosa. This breakthrough offers new hope for targeting gene therapies for the condition.
A team of Chinese and UK researchers has identified superoxide dismutase 1 (SOD1) as a potential target for reversing drug resistance in ovarian cancer. By using nanoparticles to deliver siRNA that reduces SOD1 levels, the study showed reduced growth and decreased resistance to cisplatin in female mice.
A new study from the University of Michigan Department of Neurosurgery and Rogel Cancer Center shows promising early results that a therapy combining cell-killing and immune-stimulating drugs are safe and effective in extending survival for patients with gliomas, a highly aggressive form of brain cancer. The treatment improved survival...
A new clinical trial published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for sickle cell disease. The therapy was shown to decrease vaso-occlusive events and increase production of fetal hemoglobin, providing relief from the painful condition.
Researchers at UCL and NIHR Great Ormond Street Hospital Biomedical Research Centre have developed a gene therapy that significantly reduces hearing loss in mice with Norrie disease. The treatment, which restores the missing norrin protein, has shown improved blood vessel structure and protected sensory hairs in the inner ear.
A new gene therapy study has identified microRNA-22 as a potential treatment for liver cancer, achieving better survival outcomes and reducing inflammation compared to the current FDA-approved drug lenvatinib. The treatment was administered via a single intravenous injection and showed no observable toxicity.
Researchers at the University of Minnesota Medical School have made a breakthrough in treating Hurler syndrome using gene therapy. The new approach produced normal enzymes from the liver that were able to sustain normal connections within specific neural networks, restoring brain connectivity.
Researchers have identified a neoplastic fusion transcript RAD51AP1-DYRK4 in luminal B breast cancer, associated with higher ki67 expression and aggressive clinical characteristics. MEK inhibitor trametinib may be effective in blocking the MEK-ERK signaling driven by this fusion.
A European gene therapy clinical trial has demonstrated the possibility of restoring expression of the UGT1A1 enzyme in patients with severe Crigler-Najjar syndrome. The treatment resulted in significant reduction in bilirubin levels and sustained efficacy in patients, offering a new hope for treating this rare metabolic disease.
Researchers found that rodents exposed to light for the first time in adulthood showed significant plasticity in their brains, challenging previous beliefs about adult brain rigidity. After a month, their brains looked similar to those of healthy controls, with organized visual responses and smaller receptive fields.
Researchers at CityU and HKUMed developed genetically modified human neural stem cells that promote neural circuit reconstruction, reduce glial scar accumulation, and enhance axon outgrowth. The therapy demonstrates potential for treating severe spinal cord injuries with functional recovery.
Researchers at Oregon Health & Science University have found that implanting a specific molecule in the brain can effectively reduce alcohol use among chronic heavy drinkers. The study showed a 90% reduction in drinking behavior in nonhuman primates, with the implanted virus permanently increasing dopamine levels and reducing cravings.
Researchers have discovered that gene therapy may prevent relapse in patients with serious alcohol addiction by altering dopamine function in the brain's mesolimbic reward pathway. The treatment involves sustained release of glial-derived neurotrophic factor (GDNF) in a specific region of the brain.
A study published in Proceedings of the National Academy of Sciences successfully reversed hearing loss in mice by activating a defective gene. The researchers found that introducing a special enzyme at a young age significantly improved hearing, with positive effects becoming less potent with time.
Researchers have made a significant discovery in gene therapy, showing that genetically engineered blood stem cells can be created while still in the bone marrow. This approach eliminates the need for chemotherapy and transplantation of corrected cells, cutting costs and improving access to critical treatments.
Researchers at NYU College of Dentistry developed a gene therapy that regulates sodium ion channel activity to alleviate chronic pain. By targeting the specific region where CRMP2 binds to NaV1.7, they were able to reduce sodium influx and quiet down neurons, providing relief from pain in cell and animal studies.
A team from Tokyo Medical and Dental University has developed a technique to improve bone regeneration over large areas in rats, using vascular endothelial growth factor (VEGF) and Runx2. The combination of these two RNAs led to better regenerative responses in bone cells than each RNA alone.
A team of scientists at Harvard Medical School has identified six chemical cocktails that can restore cellular aging and rejuvenate human cells. The study builds upon the discovery of Yamanaka factors, which can convert adult cells into induced pluripotent stem cells, raising hopes for treating age-related diseases and injuries. The im...
Hematopoietic stem cell culture technology improves genome editing in HSCs by increasing successful correction rates to 100%, eliminating genetic mutations, and enhancing cell transplantation outcomes. This breakthrough enhances the efficiency and safety of gene editing in treating genetic diseases.
A team of researchers has identified a unique genetic signature in CAR T-cells that enables them to persist in the body for a longer time, leading to improved remission rates for children with leukaemia. This discovery provides a new understanding of why some CAR T-cells last longer and can help improve treatment outcomes.
Researchers developed a new method to deliver drugs into the inner ear, using the cerebrospinal fluid flow. This innovation restored hearing in deaf mice and may pave the way for gene therapy to treat human hearing loss.
Researchers at MD Anderson Cancer Center have identified disparities in end-of-life immunotherapy treatment, highlighting the need for further examination to ensure quality care. A new study also reveals a novel target to improve immunotherapy responses in KRAS-mutant lung cancer and strategies to manage immune-related toxicities.
Researchers at MD Anderson Cancer Center have engineered a new model of aggressive renal cell carcinoma, highlighting molecular targets and genomic events that trigger chromosomal instability. The loss of interferon receptor genes plays a pivotal role in allowing cancer cells to become tolerant of chromosomal instability.
Researchers at Tel Aviv University have developed an innovative gene therapy that shows promise in treating Dravet syndrome, a severe developmental epilepsy affecting children. The treatment was found to be effective in improving epilepsy, protecting against early death, and enhancing cognitive abilities.
New research suggests optogenetics could restore regular heart rhythm without shocks, improving prognosis and quality of life for AF patients. Light therapy has shown promising results in rats, indicating potential translatability to humans.
Researchers at Washington University School of Medicine developed a catalog to classify protein variants associated with limb girdle muscular dystrophy. The approach enables doctors to determine the pathogenicity of variants, leading to more precise diagnoses and potential therapies.
Indiana University School of Medicine researchers will investigate muscle-directed gene therapies and test alternative treatment options for degenerative disorders like Duchenne muscular dystrophy. The goal is to develop more successful and long-term ways to help patients living with muscle disorders.
A single dose of anti-Müllerian hormone gene therapy can induce long-term contraception in female domestic cats by suppressing ovarian follicle development and ovulation. The treatment maintained high AMH levels for over two years, demonstrating its safety and effectiveness.
Researchers identified mRNAs and long non-coding RNAs targeted by stress granule proteins, which accumulate AD-associated gene transcripts in these structures. SGs may play a key role in regulating AD development through the impairment of protein neurohomeostasis.
Researchers discovered that ZC3H11A is essential for regulating metabolic genes in embryos and their absence leads to complete lethality. The study's findings suggest that ZC3 may be a promising therapeutic target for the development of anti-viral agents against medically significant human viruses.
An observational study of 329,000 Medicare admissions found that older persons receiving hospital care from allopathic (M.D.) or osteopathic (D.O.) physicians experience similar quality and cost of care. Researchers also highlight systemic health inequities faced by persons with sickle cell disease.
Researchers successfully demonstrate AAV vector efficacy in aged animal models, showing robust hearing rescue in mice with a mutation equivalent to a defective human gene. The study suggests that virally mediated gene therapy could potentially treat genetic hearing loss, especially for patients diagnosed at advanced age.
Researchers have discovered that HER3 plays a crucial role in promoting cell survival in metastatic colorectal and pancreatic cancer. The surrounding liver microenvironment activates HER3, making it an emerging therapeutic target for these types of cancer.
A new study demonstrates that subretinal delivery of AAV-RPE65 gene supplementation increases RPE65 isomerase activity and recovers chromophore 11-cis retinal after bleaching in mice with D477G RPE65 mutation, providing initial proof-of-principle data for gene supplementation as a treatment.
Increased expression of Musashi 1 on breast cancer cells has significant implications for understanding dormancy and survival in bone marrow. Msi 1 knockdown led to a reduction in cancer stem cells with undetectable PD-L1, suggesting a potential therapeutic target.
Gene therapy is being tested for rare skeletal dysplasia, affecting 1 in 200,000. Patients will receive a one-time infusion of viral vector containing correct gene sequence.
Researchers used cancer proteomics data to identify gene candidates for therapeutic targeting, focusing on protein kinases in uterine endometrial cancer cells. Public molecular resources and multi-omics data analysis can prioritize genes of interest for future studies.
Researchers developed a size exclusion chromatography (SEC) method to measure empty capsids in gene therapy programs, outperforming other analytical methods. The study found SEC-MALS to be linear, accurate, and precise, providing a robust analytical technique for advancement of gene therapy programs.
Scientists have developed a new method to deliver genetic information to stem cells using nanoparticles coated with a specific polymer, enabling more efficient control over cellular differentiation. This innovation has the potential to improve the efficiency and effectiveness of regenerative medicine treatments.
The LY6 gene family has been found to be overexpressed in uterine corpus endometrial carcinoma (UCEC), leading to poor patient survival. Several LY6 genes have been identified as potential tumor-associated antigens and biomarkers for UCEC detection and prognosis.
Researchers successfully delivered stabilized divalent siRNA molecules to animal models that blocked SARS-CoV-2 and prevented infection. The technology is adaptable for other pulmonary diseases such as pulmonary fibrosis and respiratory viruses.
Researchers identified three novel dual-purpose therapeutic targets using PandaOmics, which could treat both aging and glioblastoma multiforme. The target hypotheses include cyclic nucleotide gated channel subunit alpha 3 (CNGA3), glutamate dehydrogenase 1 (GLUD1) and sirtuin 1 (SIRT1).
Researchers at Brigham and Women's Hospital have identified a potential therapeutic target and developed a unique delivery system to treat osteosarcoma. MicroRNA nanoparticles delivered locally using a hydrogel suppressed osteosarcoma growth while decreasing bone damage.
Researchers evaluated six AAV vectors in 12 preclinical liver models to identify the most relevant combination for safe and efficient gene therapy. The study proposes using multiple models to provide a more complete picture of vector function, informing clinical decisions.
A new therapy delivery method, using modified viruses engineered with fusogens Myomaker and Myomerger, shows promise as a treatment for Duchenne muscular dystrophy. The vector can deliver a vital gene needed for muscle function to cells, potentially providing a lifelong supply of the missing gene.
Scientists design a mini gene therapy to replace the mutated gene in Usher 1F, increasing protein production and restoring hearing in mice. The approach may also prevent blindness by targeting a different form of the protein involved in vision loss.
A new genetic therapy, BIIB080, has been shown to safely lower levels of the harmful tau protein in patients with Alzheimer's disease. The trial found a greater than 50% reduction in tau protein concentrations after 24 weeks in treatment groups.
Researchers have developed a bioengineered AAV3B capsid variant with improved transduction to human liver cells and reduced seroreactivity to human serum samples. The variant, AAV3B-V04, demonstrates enhanced hepatocyte tropism and immune evasion, making it a promising candidate for gene therapy.
Cedars-Sinai investigators have discovered a novel way to treat amyotrophic lateral sclerosis (ALS) and retinitis pigmentosa using human induced pluripotent stem cells. The new approach uses cells derived from iPSCs that are renewable, scalable, and can delay disease progression in rodents.
A novel gene therapy approach decreases intraocular pressure in pre-clinical models of glaucoma. The treatment uses a viral vector to deliver instructions to cells, producing an enzyme that helps reduce fluid accumulation and pressure. This breakthrough holds promise for patients with glaucoma.
Researchers have created a drug-like cocktail that successfully regenerated hair cells in a mouse model by reprogramming genetic pathways within the inner ear. The approach could lead to clinical trials for a gene therapy that can be administered to people with hearing loss, potentially treating up to 90% of cases.
Researchers have built a new model to examine Usher Syndrome, a leading cause of combined deafness and blindness. The model replicates the visual problems not addressed by previous models, offering insight into strategies for designing therapeutic interventions.
Researchers at Tokyo Medical and Dental University have found that a specific lipid, alpha-tocopherol, increases the uptake of antisense oligonucleotides in the stroke-lesioned brain. This delivery method has potential for targeted protein expression after a stroke.
Researchers at the University of Tokyo have discovered the 3D structure of TnpB, a protein involved in genome editing and a probable precursor to the CRISPR-Cas12 enzyme. The study reveals how TnpB recognizes and cuts DNA using a unique pseudoknot shape similar to that found in guide RNAs of Cas12 enzymes.
Researchers at Children's Hospital of Philadelphia discovered that viral proteins use phase separation to coordinate the complex process of replicating viral genomes and then encapsulating them in a viral particle. This process allows for the orderly and coordinated formation of infectious viral offspring.
Researchers at Indiana University School of Medicine investigated the protein BVES and its crucial role in muscular dystrophy. They discovered that inhibiting proteasome degradation with Bortezomib can alleviate symptoms of BVES-deficient muscular dystrophy.