Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
In a groundbreaking study, gene therapy injection in one eye significantly improved vision in both eyes, with 78% of patients experiencing significant visual improvement. The treatment has shown promise in treating Leber hereditary optic neuropathy (LHON), a blinding condition affecting approximately 1 in 30,000 people.
A phase 3 clinical trial involving 37 patients showed sustainable improvements in vision after 96 weeks, suggesting the gene therapy could be a safe and effective treatment for Leber hereditary optic neuropathy. The treatment unexpectedly seemed to work in both eyes, with DNA from the vectors found in both treated and untreated eyes.
Researchers at Massachusetts General Hospital found that adding exercise to a genetic treatment for myotonic dystrophy type 1 reversed fatigue in mice. Exercise alone provided some benefit, whereas the treatment alone did not.
Scientists from Trinity College Dublin have developed a new gene therapy approach that successfully protected the visual function of mice with dysfunctional mitochondria. The treatment also improved mitochondrial performance in human cells with OPA1 gene mutations, offering hope for treating diseases like Alzheimer's and Parkinson's.
A novel targeted therapy, POMHEX, has been developed to block metabolic pathways in brain cancer cells with specific genetic defects. The study found that the small-molecule enolase inhibitor effectively killed brain cancer cells missing ENO1, and showed promise in animal models of this type of cancer.
A new consortium, Accelerating Research and Innovation for Advanced Therapies (ARDAT), aims to develop standardized models for predicting ATMP immunogenicity in humans. The €25.5 million project will also build understanding of ATMP drug metabolism within a host.
A case of in-stent thrombosis (IST) occurred in an 81-year-old man undergoing coronary bypass grafting, highlighting the complexity of antiplatelet therapy choice and genetic testing in IST risk evaluation. The patient experienced IST after converting from ticagrelor to clopidogrel therapy.
The NIH Platform Vector Gene Therapy (PaVe-GT) project uses AAV9 as a platform vector to develop gene therapy products for four rare diseases. The project aims to improve the delivery of therapeutic genes into target cells, paving the way for access to gene therapy for patients with difficult-to-treat conditions.
Researchers identified a non-hereditary mutation in blood cells from a patient with GATA2 deficiency that may have prevented bone marrow failure and other clinical manifestations. The mutation acted as a kind of natural gene therapy, protecting the patient from developing typical symptoms.
Researchers have developed a novel neuroregenerative gene therapy that converts glial cells back into functional neurons, reversing glial scar tissue. This treatment also promotes brain recovery in mouse models of ischemic stroke and Huntington's disease, offering a promising solution to the glial scar problem.
Recent studies report increased risk of rAAV mobilization in gene therapy, raising concerns for treated individuals and unintended populations. The research highlights the potential for rAAV vector production to replicate under certain conditions.
A study published on Frontiers in Cell and Developmental Biology found successful neural regeneration from brain internal glial cells for stroke repair in rhesus macaque monkeys. This gene therapy approach regenerates new neurons while also protecting injured neurons from secondary damage.
Researchers have developed a safer, more targeted way to deliver CRISPR gene therapy using light-activated liposomes. The new method uses spherical nanostructures of fat molecules to carry CRISPR molecules to specific sites in the body.
Researchers have developed a new approach to prevent toxicity seen in sensory neurons of dorsal root ganglia after gene therapy to treat neurological disorders. The approach involves modifying a transgene with a microRNA target, which reduces transgene expression and eliminates toxicity.
A new gene therapy project at the Netherlands Institute for Neuroscience seeks to repair brain damage caused by MS by identifying molecules that stimulate myelin and axon repair. The goal is to develop a drug promoting functional recovery of the nervous system, potentially revolutionizing treatment for advanced MS.
Researchers found that antiangiogenic therapy can increase the invasiveness of kidney tumors and induce a poor prognosis. A new biomarker, ALDH1A3, has been identified to predict patient response to treatment.
Researchers at Yale University screened hundreds of millions of cells exposed to COVID-19 and MERS viruses, identifying dozens of genes that enable or inhibit viral replication. The study found pro-viral and anti-viral genes, which may help predict severe illness and inform treatment development.
Researchers create genetic sensors that can detect gene activity, not just presence, using CRISPR-Cas13 system. This innovation has potential for biotech applications, including therapeutics and diagnostics.
Researchers develop reproducible protocol to assess immune effects of nucleic acid nanoparticles, a promising area of biomedical treatment. The protocol measures both quantitative and qualitative aspects of the immune response, enabling more accurate predictions and potential therapeutic uses.
A new study examines the risk of acute kidney injury among Black individuals with sickle cell trait and disease, finding a higher risk for acute kidney injury and faster loss of kidney function. The study adds important information to sparse data on the risk of acute kidney injury in those carrying the sickle cell gene.
A new study by UCLA researchers found that antiretroviral therapy given over two years was unable to completely restore age-appropriate epigenetic patterns in HIV-infected adults. This suggests that even successfully treated HIV-infected individuals are at an increased risk for early development of diseases commonly associated with aging.
A new gene therapy using a novel light-sensing protein has restored significant retinal function and vision in blind mice. The therapy involves attaching the MCO1 opsin to retina bipolar cells using gene therapy, allowing treated mice to navigate mazes and detect changes in motion faster than untreated mice.
Researchers at Mayo Clinic have developed a potential test for Machado-Joseph disease, a rare neurodegenerative disorder with no cure. The new test can quantify mutant protein levels in human biofluids, which may help evaluate the effectiveness of therapies aimed at reducing protein accumulation.
A UCI-led study demonstrates the therapeutic potential of base editing for treating inherited ocular diseases, restoring visual function to near-normal levels. The new CRISPR technology overcomes previous barriers, enabling precise and predictable correction of point mutations.
A new study has identified three gene variants that may help predict which children with eosinophilic esophagitis (EoE) are likely to respond to proton pump inhibitor (PPI) medication therapy. The research found that children carrying these variants are at increased risk of relapse after one year of PPI maintenance therapy.
Researchers create an artificial C-to-U conversion system using APOBEC1, allowing for the restoration of mutated genes and potentially treating genetic disorders. The system was tested on blue fluorescent protein (BFP) RNA with a 199T>C mutation, showing high editing efficiency.
University of Texas at Dallas researchers have identified the source of fluctuations in gene expression between identical cells, finding that transcription is the primary driver of cellular noise. This discovery has significant implications for developing targeted therapies and gene therapies.
Researchers at Kanazawa University discovered that thymoquinone selectively kills prostate cancer cells with a SUCLA2 gene deletion. This finding offers a promising therapeutic option for advanced prostate cancer patients who have developed resistance to hormone therapy.
A young woman with kaposiform lymphangiomatosis was treated with a MEK inhibitor, resulting in complete resolution of symptoms and remodeling of her lymphatic system within months. The treatment showed promise for other patients with lymphatic disorders.
Research reveals genetic differences in fat storage and formation affecting disease susceptibility in men and women. Six genes were identified as potential therapeutic targets for distinct outcomes in each sex.
Researchers identified 182 genes that regulate interaction between cancer and immune T cells, which could be harnessed for effective immunotherapy across different tumour types. The study also revealed the need for new therapy to account for genetic mutations in cancer cells that can make disease worse in response to treatment.
Researchers successfully used gene therapy to overcome cardiac effects of Friedreich's ataxia in a mouse model, achieving exercise performance similar to healthy littermates. The treatment delivered the frataxin gene via adeno-associated virus (AAV) and showed promising results.
Scientists accelerated vision restoration by visualizing photoreceptors' response to light in the living eye. Researchers modified OCT technology to detect minute changes, enabling high-resolution visualization of cone photoreceptors and potential therapies like stem cells or gene therapy.
Researchers at Children's Medical Research Institute have discovered a reason behind the low success rate of gene therapy targeting liver diseases using Adeno-associated virus 2 (AAV2). The team found that AAV2 binds too tightly to its attachment receptor, heparan sulfate proteoglycans, which leads to the vector getting
Researchers have found that specific DNA variants can affect the response to interferon-alpha-based treatments, leading to personalized therapy options. The discovery may improve treatment outcomes for patients with polycythaemia vera, a rare chronic malignant blood disease.
Capmatinib shows high response rate (68%) in first-line treatment for NSCLC patients with MET exon 14 skipping mutations. Patients with lower MET amplification levels have limited effectiveness with the drug.
A new trial suggests that children with asthma may benefit from personalized treatment based on genetic differences. The study found that children with two copies of a specific gene variant experienced an average improvement in quality of life score of 0.42.
Researchers created a system that uses CRISPR to briefly suppress genes related to AAV antibody production, allowing the virus to deliver its cargo unimpeded. The study shows promise for improving gene therapy's effectiveness and preventing or treating sepsis in mice.
A meta-analysis of NHP studies reveals that AAV gene therapy often causes DRG pathology with no clinical effects, prompting preclinical safety evaluations before clinical trials. The study's findings have the potential to streamline the development process for new vectors.
Researchers have developed a novel gene therapy approach that activates genes with similar functions to compensate for the primary defect in retinitis pigmentosa. This method has shown promise in slowing down retinal degeneration and improving retinal function without side effects.
A study by University of Pennsylvania researchers describes an effective gene therapy for Krabbe disease in dogs, which has shown promising results with no significant symptoms after four years. The treatment approach, using the AAV9 vector, has also been found to positively affect both central and peripheral nervous systems.
Researchers have discovered new genes that play a crucial role in determining the success of radiotherapy in cancer treatment. These genes, which encode for repair mechanisms used by cancer cells, can be missing or dysfunctional in some patients, making them more likely to respond to radiation therapy.
A new clinical trial, TAILOR-PCI, found that genetic testing can identify patients who may benefit from alternative antiplatelet therapy. The study showed a statistically significant 40% reduction in blood clotting events in patients with the CYP2C19 gene variation who received genotype-guided treatment.
A new study published in Pediatrics reports positive safety and early outcome data from 21 children with spinal muscular atrophy treated with gene therapy. The treatment, onasemnogene abeparvovec-xioi, is shown to be effective through age 2 years with proper screening and monitoring, but requires closer attention for potential liver is...
Scientists have engineered a tiny therapeutic delivery system using lipid-based nanoparticles that safely solve genetic problems in mice. The treatment successfully produced a protein to treat hemophilia and reduced gene activity to lower cholesterol levels, offering hope for targeted therapies.
Researchers at the University of Tokyo have shown that a treatment enhancing neuromuscular junction (NMJ) formation can improve motor function and muscle strength in aged mice. The study's findings suggest that this approach may be effective in treating age-related motor impairment and muscular weakness.
A McGill-led study reveals that suppressing the OSMR gene can improve radiation therapy effectiveness and expand lifespan in preclinical mouse models. Glioblastoma's resistance to therapy is overcome by starving cancer stem cells with energy production halted.
A targeted therapy called rucaparib has been successfully used to treat men with advanced prostate cancer who have mutated BRCA1/BRCA2 genes. The treatment resulted in a 41% objective response rate and improvements in prostate-specific antigen levels for over half of the patients.
Researchers developed novel variants of adeno-associated viral (AAV) capsids with improved transduction properties in the mouse retina and cornea. The efficient gene delivery of these variants was confirmed in non-human primate tissue, adding to their potential use in treating human ocular diseases.
A Singapore team identified a mutation in the PD-L1 gene as a reliable biomarker for patients with natural-killer T-cell lymphoma who are likely to respond positively to anti-PD-1 therapy. The study found an overall response rate of 47% and complete responses in 7 out of 19 patients.
A breakthrough study using gene therapy to target the inner retina has prevented blindness in a mouse model of CLN3 Batten disease. The treatment led to significant survival of bipolar cells and preserved retinal function, according to researchers.
Researchers at CNIO develop effective therapy for mice with age-related pulmonary fibrosis, activating telomerase production to prevent disease development. The treatment, a gene therapy, also successfully reversed fibrosis in mice without genetic alterations.
Researchers have discovered a promising new treatment strategy for patients with prion disease by reducing levels of prion protein. This approach has been shown to significantly extend the survival of lab animals and even reverse disease markers after toxic clumps have formed in the brain.
Researchers developed a nanomedicine-based strategy for chemo-immunotherapy that eradicated PTEN-negative glioblastoma cells. The combination of epirubicin-encapsulating nano-micelles and immune checkpoint inhibitors increased tumor-infiltrating T cells and reduced immunosuppressive cells, effectively killing cancer cells.
Dr. Sanjana's research uses high-throughput genome engineering technologies to identify genes that can boost the effectiveness of CAR T-cell therapy for pancreatic ductal adenocarcinoma. The project aims to overcome immunosuppression in pancreatic cancer and potentially lead to improved immunotherapies.
Researchers at the University of Pennsylvania have identified a new genetic cause of Charcot-Marie-Tooth disease, a debilitating neurodegenerative disorder that affects nearly one in two thousand people. The discovery sheds light on the genetic underpinnings of the disease and may hold answers to new gene therapies.
A new therapeutic approach for CLN3 Batten disease has been developed by a team led by Rosalind Franklin University researcher Michelle Hastings. The treatment uses antisense oligonucleotides to target the mutated gene causing the disease, showing promise in rodent models and human cells cultured from patients.
Researchers at the Wellcome Sanger Institute have engineered a new mouse model to study glioblastoma, the most aggressive type of brain cancer. The study identified over 200 genes that contribute to the development and growth of glioblastoma, providing potential new drug targets.
A study has identified a genetic synergy that can cause cancer cell death, which can be replicated by a drug-like molecule and exploited for therapy. The findings suggest that FEN1, a gene involved in DNA replication and repair, has synthetic lethal interactions with BRCA-mutant cells.
A team of researchers used a mouse model to test the best therapeutic option for a type of relapse sarcoma. The study found that two treatment regimens administered to a patient with lung metastasis helped extend their disease-free period to 46 months.