Articles tagged with Gene Therapy
A new study reports on five patients with Canavan disease who have a novel variant identified through targeted long-read sequencing, revealing an SVA_E retrotransposable element that disrupts gene function. The findings enhance genetic diagnostics and enable improved guidance for families.
A recent study published in the New England Journal of Medicine found that gene therapy can significantly reduce the risk of severe motor and cognitive impairment in children with metachromatic leukodystrophy (MLD). The therapy, administered early, preserves motor function and cognitive abilities in most patients.
A UC Riverside-led study found that adult stem cells rely on histone chaperones to maintain their regenerative capacity. The researchers discovered that disrupting these proteins can lead to specific changes in stem cell identity, potentially guiding them into desired cell types.
Novel biomarkers like miRNA-34a link anthracyclines to cardiotoxicity, while stem cell therapy and nanotechnology offer potential for prevention and treatment. Traditional strategies have limitations, but new approaches hold hope for improved patient outcomes.
A team of researchers has developed a gene-therapy strategy to treat arrhythmogenic right ventricular cardiomyopathy type 5 (ARVC5), a rare and deadly hereditary disease. The treatment, using adeno-associated viruses, improved cardiac function and prolonged survival in mice with ARVC5-like disease.
A recent trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, with the immune system emerging as a key barrier to its success. Researchers identify early intervention at birth and personalized immune screening as potential solutions to overcome this hurdle.
Researchers found that NSD2 helps maintain MM cell identity by reorganizing DNA and influencing gene activity. This discovery could shape future treatment approaches for patients with t(4;14) myeloma.
A multidisciplinary team has generated an atlas to optimize gene therapy delivery, providing researchers with insights into the most effective viral vectors for specific tissues. The study identifies AAV4 as a promising vector for vascular and pancreatic applications, offering new possibilities for treating conditions like diabetes.
A recent study demonstrates gene therapy's potential to restore motor capacity in an ultra-rare disease, Megalencephalic Leukoencephalopathy with Subcortical Cysts (MLC), even after symptom onset. The treatment restored normal protein activity, normalizing physiological brain alterations and reversing motor impairments in treated mice.
Researchers at MD Anderson Cancer Center made several key discoveries, including the spatial organization of cancer-associated fibroblasts across various cancers and a study on treatment resistance in SMARCA4-mutant lung cancer. These findings highlight the importance of investigating cell populations in their spatial context to better...
Researchers at the NIH developed eye drops containing a short peptide derived from PEDF, which slowed photoreceptor degeneration and vision loss in animal models of retinitis pigmentosa and dry age-related macular degeneration. The treatment preserved up to 75% of photoreceptors and showed no negative side effects.
Scientists have developed a new gene replacement therapy in mice that alleviated symptoms without side effects, offering long-term recovery for patients. The therapy targets the SCN1A gene, which plays a crucial role in regulating brain activity, and shows promising results in treating Dravet syndrome.
Researchers at Rice University have gained insights into ADAR1's molecular mechanisms, which could lead to improved treatments for cancer and autoimmune diseases. The study found that ADAR1's editing activity depends on RNA sequence, duplex length, and mismatches near the editing site.
Researchers have developed a new gene therapy protocol using the SIN-EFS-IL2RG.co vector, which demonstrates safety and efficacy in preclinical studies. The treatment restores immune functions and lacks oncogenicity, paving the way for further clinical trials in X-SCID patients.
Researchers have discovered new CRISPR-Cas systems with improved efficiency and specificity, including one from dairy cow bacteria that can target specific gene sequences. The new systems have potential applications in human health, biotechnology and environmental fields.
Researchers are conducting a first-in-human clinical trial to test a modified herpes virus that targets spinal cord nerve cells to treat neurogenic bladder. The therapy, EG110A, aims to block sensory nerve signals causing involuntary bladder contractions and incontinence.
Researchers discovered a molecular switch, FLI-1, essential for blood stem cells to enter an activated state. Transiently producing FLI-1 in quiescent adult mobilized bone marrow stem cells activates them, improving their ability to expand and restore the blood cell supply in a new host.
The new AI model leverages hypergraphs to quickly and accurately identify therapeutic gene targets for diseases. HIT outperformed existing models in all tested metrics, demonstrating its accuracy in classifying therapeutic gene targets with great precision.
A new study reveals that radiotherapy has opposite effects on glioblastoma multiforme (GBM) and low-grade gliomas (LGG), with GBM patients living longer after treatment. The study highlights the need for personalized treatment approaches based on genetic and molecular characteristics to improve survival outcomes.
A gene therapy treatment has demonstrated significant promise in treating maple syrup urine disease (MSUD), a rare genetic disorder. The therapy prevented death and normalized growth in animal models, including a calf with the condition, and is being explored for potential use in humans.
Scientists discovered a protective variant of the HAQ-STING gene that prevents COPA Syndrome. This finding opens the door to a new gene therapy for the condition, which currently has no cure.
A novel bone marrow transplant process has been shown to be safe and curative for adults with sickle cell disease, offering a viable alternative to recent gene therapy products. The treatment, which uses a 'half-matched' donor, results in high cure rates and low side effects, making it a more accessible option for patients.
A new genetic medicine has been developed to treat a rare genetic deficiency affecting the AIPL1 gene, causing severe retinal dystrophy. The treatment involves injecting healthy copies of the gene into the retina through keyhole surgery, resulting in dramatic improvements in sight for four young children.
Researchers have discovered a novel gene therapy that can reverse conduction slowing and prevent cardiac arrhythmias by introducing the SCN10a-short gene into heart muscle cells. The treatment has shown promise in animal models and human cell studies, offering a potential solution for millions affected by arrhythmias worldwide.
Researchers at Virginia Tech have developed a method to convert gut bacteria into mini protein factories that produce and release sustained flows of targeted proteins within the lower intestine. This approach eliminates a major roadblock in delivering drugs to this part of the body, offering potential treatment for chronic diseases.
A new gene therapy has shown promise in treating a rare form of epilepsy, specifically Dravet syndrome, by replacing the SCN1B gene variant. The therapy increased survival rates, reduced seizure severity, and restored brain neuron excitability in mice with the condition.
The Rice University lab, in collaboration with Baylor College of Medicine, has developed a new gene-editing strategy called Repair Drive that improves the effectiveness of gene therapies in the liver. The technique enables the repair of liver cells at higher rates and equips them with a selective advantage to outcompete incorrectly edi...
Researchers at Virginia Tech have developed a way to convert gut bacteria into miniature protein factories that produce and release targeted proteins inside the lower intestine. This breakthrough could potentially treat chronic diseases.
Researchers discovered that mismatch repair genes are critical in eliciting damages to neurons vulnerable to Huntington's disease, triggering downstream pathologies and motor impairment. Targeting these genes may offer novel therapeutic approaches, including improving locomotor and gait deficits and reducing neuronal cell death.
Researchers at Osaka Metropolitan University assessed target genes in canine hepatocellular carcinoma (HCC) to develop molecular targeted therapies. The study identified potential gene targets, including PDGFB, which may improve treatment options for unresectable HCC.
Dr. Eric Levine's lab at UConn School of Medicine has been awarded $400,000 to study the genetic pathways of autism spectrum disorders using patient-derived human neurons. The team aims to identify genes and brain cell behavior that may cause these disorders, with potential applications for gene therapy.
Researchers developed a novel AAV-equipped nanomachine that successfully overcame gene therapy challenges in mice, including reduced efficiency due to neutralizing antibodies and hepatotoxicity. The nanomachine demonstrated sufficient gene transfer activity and suppressed liver toxicity markers.
A new study adds weight to the safety and effectiveness of a gene therapy for hypophosphatasia, a rare inherited disorder that causes abnormal bone development. The treatment, AAV8-TNAP-D10, has shown promising results in mice models, with female mice achieving improvements in bone and teeth at lower doses.
Researchers have developed a new gene therapy that targets aggressive brain cancer, glioblastoma, with a precise delivery system. The treatment uses a novel virus to deliver a targeting drug to cancer cells, achieving cure rates of up to 90% in mouse models.
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.