Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
Researchers studied the effects of resveratrol on circadian clock gene expression in young and older human adipose-derived progenitor cells. They found increased levels of some components in older-APCs compared to young-APCs, but also observed gained rhythmicity of some components after resveratrol treatment.
Researchers have successfully used mRNA technology to correct a rare genetic disease in mice, demonstrating its potential therapeutic use. The treatment corrected the lethal consequences of the disease and restored glutathione metabolism.
Rice bioengineer Jerzy Szablowski and colleagues have engineered a synthetic serum marker that enables non-invasive neural monitoring by tracking gene expression dynamics in the brain. This breakthrough allows researchers to investigate brain development, cognitive function and neurological diseases more effectively.
Researchers at Baylor College of Medicine have developed a technology to effectively regulate gene expression, promising a safer approach to gene therapy. The system uses small molecules to interact with RNA, allowing for precise control over protein production and therapeutic window maintenance.
Researchers at UCL Queen Square Institute of Neurology have developed a new gene therapy that significantly reduces seizures in mice with focal cortical dysplasia. The treatment, which involves the overexpression of a potassium channel, has shown an average reduction of 87% in seizures without affecting the mouse's memory or behavior.
Researchers developed a gene therapy approach to target the root cause of ACM, restoring plakophilin-2 levels and improving heart function. Clinical trials starting in 2024 aim to explore this approach in patients with PKP2 mutations.
Gene therapy treatments can prompt an adverse immune reaction when the body mistakenly perceives the treatment as a viral threat. Researchers have identified a critical pathway in the liver that triggers this response, but also found a way to block it, paving the way for safer and more precise gene therapy options.
A team of researchers has developed a promising gene-editing strategy for spinal muscular atrophy (SMA), a devastating pediatric neuromuscular disorder. The approach involves using CRISPR base editing to activate the SMN2 gene, which is similar to the mutated SMN1 gene responsible for SMA.
Researchers at UC Davis Comprehensive Cancer Center have shown that inhibiting galectin 1 protein using gene therapy can shrink liver cancer in mice, improving the anti-cancer immune response and increasing killer T cells. This approach has potential as a treatment for HCC prevention.
Researchers at NTNU are developing a new therapy for rare hereditary diseases like DOOR syndrome using mini-brains grown in the lab. The treatment involves injecting a virus with a healthy OXR1 gene to produce the missing protein, which may help stop or reverse the disease.
Researchers discovered a link between gene therapy and the accumulation of stem cells with genetic mutations, which can lead to accelerated growth and increased blood cancer risk. The study suggests that younger patients may be safer candidates for gene therapy due to fewer genetic mutations.
A new review article highlights significant milestones in gene therapy for rare diseases, improving efficacy and safety. However, challenges remain to make this transformative approach widely available.
The Genes & Health study has enrolled its 10th participant in a gene-editing clinical trial for heart disease, specifically familial hypercholesterolemia. This milestone marks an important step towards improving health outcomes for people of Pakistani and Bangladeshi descent.
Researchers at Duke University developed a CRISPR-based platform to identify genes that improve T-cell therapies for cancer treatment. They discovered BATF3, a single master regulator of the genome, which reprograms thousands of genes in T cells and greatly enhances cancer cell killing.
Researchers created an immunodeficient mouse model of physiological ovarian aging, mirroring female fertility decline with age. The study found decreased oocyte quality, impaired follicle activation and growth, and increased mitochondrial dysfunctions in Old mice.
A novel gene therapy strategy has been developed to specifically target and activate the direct pathway in Parkinson's disease-affected neurons, leading to improved motor symptoms such as bradykinesia and tremor. The approach has shown faster onset and longer duration compared to traditional L-Dopa treatment.
Individuals with severe sickle cell disease express willingness to accept gene therapy risks for a potential cure. Gene therapies offer higher chances of eliminating symptoms and extending life expectancy, but also come with significant risks.
Researchers have developed a novel approach, REVeRT, to efficiently transport large genes using dual AAV vectors at the transcript level. This new method offers increased efficiency, fewer side effects, and greater flexibility compared to existing strategies.
Researchers developed the DANGER analysis tool to overcome challenges in CRISPR gene editing. The tool assesses on- and off-target effects without a reference genome, quantifying phenotypic risk at the gene ontology level. This breakthrough enables safer strategies for genome editing in various organisms.
Researchers at the University of Texas Health Science Center discovered a gene therapy approach that can restore motor functions in mice with Contactin-Associated Protein 1 (Cntnap1) mutations. The study found that turning on the normal gene earlier improves the rescue outcome, and the next phase is to test this approach in humans.
In a phase I trial, an oncolytic virus treatment designed by Brigham researchers extended survival among patients with recurrent glioblastoma, especially those with pre-existing viral antibodies. The therapy reshaped the tumor's surrounding environment to stimulate an anti-tumor immune response.
Researchers identified the integration site pattern of lentiviral gene therapies in patient cells, shedding light on treatment safety and efficacy. The study found that integration near nuclear pores is associated with improved safety and effectiveness, avoiding oncogene regions where earlier technologies failed.
Researchers used CRISPR gene editing to study the impact of CHD8 mutations on human cortical neurons. The study found that CHD8 alterations drive molecular and cellular defects in neurons, leading to reduced activity and synaptic communication.
The Keck School of Medicine of USC has received a $2 million grant from the California Institute of Regenerative Medicine to further enhance its cutting-edge cGMP Laboratory. The funding will support the adoption of advanced technologies, including an electronic quality management system and optimized cell therapy manufacturing processes.
USC is partnering with seven leading regenerative medicine institutes to form the Los Angeles and surrounding area regenerative medicine consortium. The partnership aims to advance regenerative medicine using stem cells and gene therapies for treating unmet medical needs.
Scientists from Tokyo Metropolitan University have created a new polymer that can effectively transport plasmid DNA into T-cells during CAR T-cell therapy. The polymer, called PAMAM-G2-Gu, is stable, non-toxic, and doesn't use viruses, making it a promising candidate for next-gen gene carriers.
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.
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.
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 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 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.
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.
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.