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 have identified ALPK2 as a potential therapeutic target for treating heart failure with preserved ejection function (HFpEF). The enzyme is believed to prevent stiff heart conditions through regulating the TPM1 gene. This discovery offers new hope for developing treatment options targeting ALPK2.
The foundation has awarded eight recipients of the 2025 Damon Runyon-Rachleff Innovation Award, including five early-career researchers with initial grants of $400,000 over two years. The awardees aim to develop novel cancer therapies using innovative approaches such as engineered skin bacteria and small molecule-boosted drug delivery.
Batten disease, a neurodegenerative disorder, affects the bowel wall's enteric nervous system, causing degeneration and bowel symptoms. Gene therapy has been shown to slow symptoms and extend lifespan in mice by preventing enteric neuron degeneration.
Researchers at Johns Hopkins Medicine identified a new epigenetic approach to target colorectal cancer, using a mouse protein that disrupts cancer-causing chemical changes in genes. The study found that the protein, STELLA, can be used to develop a drug strategy to treat solid tumors.
Researchers developed a gene-editing treatment that reduces prion protein levels in the brain by up to 60% and extends mouse lifespan by about 50%. The base-editing approach could lead to a one-time treatment for prion disease patients.
Researchers investigated how two CAR T cells kill cancer with distinct signaling domains. CD28.ζ-CAR molecules work quickly and efficiently, while 4-1BB.ζ-CAR molecules linger in lipid rafts for sustained collaborative killing of tumor cells. This study aims to design CAR molecules maximizing antitumor activity beyond B cell malignancies.
Researchers have identified a 177-gene signature common to metastasis across cancers, allowing for personalized risk assessment and potential therapies. The discovery could lead to broader treatment options, faster drug access, and improved patient outcomes.
Researchers at UTHealth Houston have discovered two novel genes, DYRK1A and EGFR, linked to genetic mutations causing epileptic brain lesions. This breakthrough offers a new framework for understanding epilepsy and developing targeted therapies.
A zebrafish protein, Hmga1, has been found to unlock dormant genes for heart repair in mice. The discovery could lead to regenerative therapies to prevent heart failure in humans.
Researchers developed AI-driven therapeutic platform mimicking viral structures to deliver therapeutic genes to target cells. The innovative approach achieved precise symmetrical structures and effectively delivered payloads, paving the way for breakthroughs in gene therapies and next-generation vaccines.
Researchers have identified molecular changes that occur long before symptoms appear, shedding light on the development of Rett syndrome. These findings hold promise for developing safe gene therapies and monitoring biomarkers to track MECP2 gene function.
Researchers aim to advance eye transplantation through cutting-edge technologies and techniques, including cell-based therapies and device development. The six-year project, led by Kia Washington at the University of Colorado Anschutz Medical Campus, seeks to overcome current limitations in whole-eye transplant success rates.
A new study shows targeted delivery of energy-disrupting gene therapy using nanoparticles shrinks glioblastoma brain tumors and aggressive breast cancer tumors in mice. The technology, mLumiOpto, induces light-activated electrical currents inside cells to disrupt mitochondria, leading to programmed cell death and DNA damage.
Researchers have developed a new generation of cell-penetrating antibodies that can target cancer cells and deliver therapeutic molecules directly into tumor cells. The 3E10 antibody shows great promise for treating cancers with defective DNA repair pathways.
A new gene therapy has reversed the effects of heart failure in a large animal model by increasing blood pumping efficiency and dramatically improving survival rates. The therapy restored critical functions of heart cells and improved heart function on the microscopic level.
Researchers have identified new potential therapeutic targets for diabetic kidney disease (DKD), a leading cause of kidney failure. The study reveals common and cell-type-specific changes caused by insulin-resistance, representing new targets for pharmacological or targeted gene therapy approaches.
Branden Baptiste, 20, becomes the world's first patient to receive base editing gene therapy for sickle cell disease. The treatment has left him feeling 'more than fine' with no symptoms of the disease. After undergoing chemotherapy and a series of tests, Branden was infused with genetically treated cells in December 2023, allowing his...
A new gene therapy has shown promise in reversing a condition that causes significant stillbirths and premature deliveries globally. The therapy, developed by University of Florida Health researcher Helen N. Jones, successfully boosted placental function and delivered normal-weight offspring in guinea pig models.
A Phase 2 clinical trial has been initiated by Genethon and Hansa Biopharma to evaluate the efficacy and safety of imlifidase, a gene therapy for Crigler-Najjar syndrome. The trial aims to address anti-AAV antibodies, which prevent up to 1 in 3 people from benefiting from gene therapies.
Researchers at UCSF are enrolling patients in a clinical trial to correct the genetic mutation causing sickle cell disease using non-viral CRISPR-Cas9 gene editing. The therapy aims to eliminate the need for a bone marrow transplant and create a new blood system free of the disease.
Positive initial results from Genethon's gene therapy GNT0004 show stabilization of motor functions and improved dystrophin expression in patients with Duchenne Muscular Dystrophy. The therapy is expected to be launched in pivotal trial phases in Europe and the US in Q2/2025.
Researchers discovered a protective inherited mutation in the ADNP gene that enhances protein interactions and offers protection against developmental disorders. The study challenges previous assumptions about genetic mutations and their impact on brain development.
A new RNA-based technology called StitchR facilitates effective use of gene therapy for difficult-to-treat, large-gene diseases like muscular dystrophies. It delivers two halves of a gene separately, resulting in seamless reconstitution of large mRNA in affected tissues.
Researchers from UAB have developed a gene therapy that reverses metabolic dysfunction-associated steatohepatitis (MASH) in mice, and most obese and type 2 diabetic patients could benefit from this treatment. The therapy, based on the fibroblast growth factor 21 protein, mediates long-term reversal of liver fibrosis and MASH.
The institute's Spark Grant program recognizes innovative research in gene and cell therapy, with recipients working on projects spanning disease fields such as neuroinflammation, cancer, and tuberous sclerosis. The $1.15M grant will support the development of novel treatments and commercialization outcomes.
More than 70 hematology researchers from the University of Miami Miller School of Medicine will showcase their work at the 66th ASH Annual Meeting & Exposition. Researchers from Sylvester Comprehensive Cancer Center are authors or co-authors on a significant number of posters presented during the event.
Researchers have developed a groundbreaking gene therapy that targets Diamond-Blackfan anemia, a rare genetic blood disorder. The treatment uses regulated GATA1 expression to correct multiple genetic mutations with a single vector, offering new hope for patients currently reliant on lifelong follow-up care and stem cell transplants.
The CRISPR tool was successfully used to correct a genetic defect in cells affected by chronic granulomatous disease. However, the repair process also introduced new genetic defects, highlighting the need for caution when using CRISPR technology in clinical settings.
The US Department of Defense has awarded $514,000 to study the efficacy of gene editors in treating Duchenne Muscular Dystrophy. Researchers will explore non-viral options for delivering gene therapy through 'self-delivering' gene editors, aiming to improve safety and efficacy.
A new study in mice shows a unique mRNA delivery method can successfully edit faulty genes in fetal brain cells. The technology has the potential to stop progression of genetic-based neurodevelopmental conditions like Angelman syndrome and Rett syndrome before birth.
The ASHG 2024 Annual Meeting will showcase the latest research in human genetics and genomics. The event will feature a Presidential Symposium on Mendelian traits and a Distinguished Speakers Symposium on the promise of human genetics and genomics, among other sessions.
Researchers aim to improve gene therapy design for life-long correction of genetic diseases, but face unknowns including immune response and genomic changes. A five-year NIH award will fund an analysis of genetic and cellular determinants of gene therapy longevity.
Researchers have identified a protein called PERM1 that regulates both energy and heart muscle contraction, offering a new therapeutic approach to systolic heart failure. By addressing the underlying problem of weakened heart muscle, PERM1 may help restore cardiac function and improve patient outcomes.
A large-scale study of children with genetic disorders found that thousands benefited from targeted treatments and support after receiving a genetic diagnosis, with over 20% able to start or adjust therapies. Researchers expect this number to grow as new genetic therapies develop.
A clinical trial of 32 patients with cerebral adrenoleukodystrophy found that six years after treatment, most remained free of major disabilities. However, the study also highlighted safety concerns about blood cancers post-treatment, including myelodysplastic syndrome and acute myeloid leukemia.
Researchers have developed a novel gene therapy approach that targets and breaks down faulty ribonucleic acids in the KCNA2 gene, which is associated with recurring seizures. The therapy has shown promise in reducing excessive neuron activity linked to epilepsy.
Researchers review UBA1 loss of function in VEXAS Syndrome, a hematoinflammatory disorder characterized by severe inflammation, cytopenias, and oncogenicity. They explore therapeutic options, including clone-targeting drugs, to combat this challenging disease.
Two UCF cancer researchers, Alicja Copik and Debbie Altomare, have received $100,000 grants from the Florida Breast Cancer Foundation to develop new treatments for breast cancer. They focus on enhancing natural killer cells to fight cancer and harnessing the body's immune system to create new therapies.
Researchers developed a novel approach to optimizing siRNA-loaded lipid nanoparticles using NMR-based molecular-level characterization. Pre-mixed LNPs exhibit superior gene-silencing effects due to a stacked bilayer structure that enhances gene silencing.
A single-dose gene therapy has been shown to significantly reduce bleeding episodes in adults with hemophilia B, with an average reduction of 71% compared to standard treatment. The therapy, which enables the liver to produce clotting factor IX, has been FDA-approved for use in patients with this genetic disorder.
Researchers discovered that DNA methylation patterns, like cellular memory markers, prevent reprogrammed cells from fully adopting new identities. This limitation limits the effectiveness of long-term treatments and therapies.
A new NIH-funded project aims to address gene therapy's ethical and policy challenges, gathering information from experts in six domains. The study will provide recommendations for addressing these challenges, with implications for patient safety and health outcomes.
Scientists have developed new therapies that selectively remove aggregated tau proteins associated with Alzheimer's disease in mice. The approach utilises TRIM21 to target tau aggregates, leaving healthy tau intact, and demonstrates potential for other brain disorders driven by protein aggregation.
Genethon has developed an innovative gene therapy vector that effectively targets muscle tissue while reducing the risk of liver penetration. The new capsid design uses AI predictive methodology to improve efficacy and safety, paving the way for more effective treatments for neuromuscular diseases.
Researchers at Boston University discovered a new method to harness self-amplifying RNA to create more effective vaccines. The modified saRNA vaccine protected mice from severe COVID-19 disease with a lower dose than current mRNA vaccines. Longer duration of protein expression and reduced inflammation were also observed.
Researchers at Karolinska Institutet successfully used gene therapy to improve vision in 11 out of 12 patients with Bothnia dystrophy, a form of hereditary blindness. The treatment involved injecting a specially designed virus under the retina, which produced normal protein and restored visual function.
Children's Hospital of Philadelphia researchers have reported encouraging evidence of an effective new gene therapy to treat multiple sulfatase deficiency. The ex vivo gene therapy improved sulfatase production and reduced symptoms associated with the disease in preclinical models.
Scientists from Spirovant Sciences describe a novel adeno-associated virus (AAV) gene therapy called SP-101 that has been optimized for efficient human airway cell transduction. After single dose inhaled delivery, the vector showed consistent expression of a functional and regulated shortened human CFTR minigene.
Scientists from Trinity College Dublin have developed a gene therapy that protects retinal ganglion cells and improves their function in animal models of glaucoma. The therapy has also been shown to increase oxygen consumption and ATP production in human retinal cells, indicating enhanced cell performance.
Researchers at Nagoya University have developed a method to chemically alter siRNAs, reducing off-target effects and improving the safety of siRNA drugs for genetic therapy. By modifying the seed region of siRNAs with formamide, they achieved suppression of off-target effects with higher efficiency than existing chemical modifications.
Researchers developed a gene therapy that restored useful vision to most patients with Leber congenital amaurosis type I, a rare inherited blindness. The treatment showed a 10,000-fold improvement in light sensitivity and improved navigation abilities in patients who received the highest dose.
Experts propose a series of scientific principles and experimental approaches to assess potential carcinogenicity of gene therapies. Data transparency is crucial, with publicly accessible data from viral integrations site studies and clinical settings.
A multi-university research team led by University of Virginia engineering professor Gustavo K. Rohde has developed a system that can accurately spot genetic markers of autism in brain images. The system uses generative computer modeling technique called transport-based morphometry, which reveals brain structure patterns that predict v...
Researchers have developed two new methods to produce circular RNAs, which can silence genes and serve as templates for making therapeutic proteins. These circular RNAs display enhanced stability and biological activity in heart muscle cells and neurons.
A recent study found that Chinese patients with ovarian cancer often carry a specific variant of the RAD51D gene, which can promote tumor growth. The variant also makes these patients more sensitive to PARP inhibitors, leading to a favorable prognosis and potential new treatment methods.
Researchers developed a treatment approach using lipid nanoparticles to deliver mRNA therapy for MSUD, extending survival and reducing serum leucine levels. The study also identified a novel AAV variant with desirable biodistribution properties for targeting peripheral organs.
Researchers at the Broad Institute of MIT and Harvard developed a machine-learning approach to design better AAVs for gene therapy. The tool helps engineer capsids with multiple desirable traits, such as targeting specific organs or working in multiple species. About 90% of predicted capsids successfully delivered cargo to human liver ...
The Rice University lab has developed a new noninvasive technique, called REMIS, that can measure gene expression and gene therapy delivery in specific brain regions using ultrasound. This technology could revolutionize brain-based gene therapy for neurodegenerative diseases.
Researchers at the University of Helsinki have created cells that can't grow uncontrolledly, which could lead to new and safer cell therapies for hereditary diseases, myocardial infarction, and other conditions. The innovation uses thymidine supplementation to regulate cell division, eliminating the risk of cancer.
β-Thalassemia patients experience iron overload due to increased intestinal absorption and transfusions, leading to oxidative stress and tissue damage. Chelation therapy is the cornerstone of treatment, but novel therapeutic strategies are needed to optimize patient outcomes.