Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
Jackson Laboratory Assistant Professor Mingyang Lu has developed a computational approach called RACIPE to capture genetic events in cellular decision-making. The algorithm could have broad impact on basic research and designing new therapeutic interventions in genomic medicine.
The Alliance for Cancer Gene Therapy (ACGT) is funding innovative clinical approaches to find gene therapy treatments for solid cancers. Recipients of the 2018 research grants include a team working on a vaccine for melanoma, an injectable genetic agent to program T cell receptors, and CAR T cell therapy for brain cancer.
Chinese researchers have improved the adenine base editing (ABE) system to generate mouse or rat models for human genetic disorders, such as DMD and Pompe disease. The ABE system allows for efficient alteration of genetic codes with minimal undesired outcomes, making it a promising technology for therapeutic applications.
Researchers developed a gene therapy that restored function to the olfactory system in genetically modified mice. The therapy increased cilia and sensory neuron responses, leading to restored connections between neurons and behaviors in odor-guided tasks.
Researchers at OHSU have discovered a naturally occurring neurodegenerative disease in monkeys that mimics Batten disease, a fatal childhood disorder. The study confirms the CLN7 gene mutation and provides a promising model for developing new gene therapies.
A new study published in Nature Medicine found that fetal gene therapy can prevent and cure neonatal lethal neurodegenerative diseases in humans in utero. The therapy was tested on mice with Gaucher disease and showed promising results, including improved brain degeneration and increased survival time. Researchers believe this approach...
Researchers from UAB successfully used gene therapy to treat type 2 diabetes and obesity in mice by inducing the production of FGF21, a hormone that maintains correct energy metabolism. The treatment led to weight loss and improved insulin sensitivity, reducing the risk of related comorbidities.
Researchers have successfully used viral vectors to deliver genetic material to kidney cells, paving the way for new treatments for chronic kidney disease. The discovery could lead to gene therapies that slow or reverse the growth of cells contributing to CKD.
Researchers at Washington University School of Medicine have developed a gene therapy method to target damaged kidney cells, which could lead to improved treatment for chronic kidney disease. The approach uses adeno-associated virus (AAV) to deliver genetic material to targeted cells, showing promise in slowing or reversing cell damage.
Researchers successfully used CRISPR-Cas9 genome editing to restore normal levels of the AAT enzyme in mouse models of alpha-1 antitrypsin deficiency. The approach showed promise for treating patients with this genetic disorder.
A new molecular imaging method allows early assessment of gene therapy success, potentially improving treatment for Parkinson's and Alzheimer's diseases. The PET reporter gene/probe system enables noninvasive monitoring of gene expression in all brain areas.
Cystic fibrosis patients may benefit from a new lipid-based nanoparticle therapy that enables cells to produce the correct protein, allowing for improved respiratory function. The therapy has shown promise in treating all cystic fibrosis patients, regardless of their genetic mutation.
Researchers found substantial overlaps in genetic risk for psychiatric disorders, including ADHD, bipolar disorder, major depressive disorder, and schizophrenia. In contrast, neurological disorders showed limited evidence of common genetic risk factors, suggesting greater diagnostic specificity.
University of Michigan researchers have made a breakthrough in treating Spinocerebellar ataxia type 3 (SCA3) using gene silencing technology. They successfully reduced mutant RNA levels without toxic effects, offering hope for disease-slowing therapy.
AcuraStem has been awarded a $3.7 million SBIR grant to continue research on a small molecule therapeutic for ALS, utilizing induced motor neuron cellular models and precision platform iNeuroRx°. The grant supports the development of AS2015, targeting expansion repeats in the C9ORF72 gene.
A new RNA-Seq assay detects gene fusions in solid tumor cells with high accuracy, identifying 93% of fusions missed by other techniques. The assay also discovers 18 novel fusions, including 11 previously unknown combinations, which may have clinical significance.
A new gene therapy has been shown to restore hand function in rats with spinal cord injuries by breaking down scar tissue and allowing nerve cell regeneration. The therapy uses a 'stealth gene' switch that can be turned on and off, providing a safeguard and paving the way for human trials.
A novel medication, BYL719, demonstrates efficacy in treating CLOVES Syndrome by reducing vascular masses and improving quality of life. Patients show significant symptom reduction, growth stabilization, and improved overall health.
A rapid test can distinguish infants with a specific genetic change from those without it, allowing tailored prescribing and preventing antibiotic-related deafness. The test uses a cheek swab and can produce results in around 40 minutes.
The study found that matching targeted therapies to tumor-specific gene mutations improved progression-free and overall survival in patients with advanced disease. Receiving matched targeted therapy was also an independent factor for predicting longer overall survival.
A novel data-driven approach called PanDrugs prioritizes cancer treatments based on patient genetic alterations. The method integrates pathway context and collective gene impact to provide therapeutic options for patients with limited druggable genes.
Researchers at Kyoto University developed a synthetic molecular code that can script gene activation, targeting histones and emulating the natural histone acetylation process. The code, called Bi-PIP, successfully activated a specific gene associated with central nervous system disorders in living cells.
Dr. Michel Sadelain shares his role in advancing CAR T-cell therapy, a rapidly evolving field that targets cancer cells. He describes the FDA approval of the first CAR T-cell therapy and predicts next-generation technology advancements.
Researchers at Michigan State University have developed a gene therapy that restored vision in dogs with progressive retinal atrophy, a condition similar to human retinitis pigmentosa. The treatment has shown a 100% success rate and is now being advanced for human use with a $8.2 million grant.
The Damon Runyon Cancer Research Foundation has announced five new Physician-Scientist Training Awards, which will provide $2.3 million in funding for the recipients. The awards aim to address the financial disincentives that often deter physicians from pursuing a research career.
Tivanisiran, an RNAi-based compound, shows promise in treating dry eye syndrome by improving ocular inflammatory parameters and reducing symptoms. The Phase III Helix study will evaluate its efficacy in over 300 patients across six countries.
A gene therapy case report published in Human Gene Therapy reveals a safe and effective treatment for lipoprotein lipase deficiency, with improved quality of life outcomes after discontinuing plasmapheresis. The treatment, ali-pogene tiparvovec, has been approved in Europe to treat the rare inherited disorder.
A study by Dana-Farber Cancer Institute and Broad Institute identified five genetic subtypes of diffuse large B cell lymphoma, which can help predict individual patient outcomes and guide personalized treatment. The analysis revealed clear links between specific genetic signatures and how patients respond to standard treatment.
Researchers have identified a therapeutic RNA molecule that corrects the genetic processing error causing familial dysautonomia by enabling human and mouse cells to produce normal levels of IKAP protein. The discovery provides proof of concept for potentially treating this rare disorder.
Researchers have developed a gene therapy approach to treat GM2 gangliosidosis, a devastating neurodegenerative disease. The treatment has shown promising results in mouse models and is now being tested in clinical trials using recombinant Adeno-associated viruses for gene transfer.
A global research project has identified 44 genetic variants linked to major depression, 30 of which are newly discovered. These findings suggest a shared genetic basis with other psychiatric disorders like schizophrenia.
Researchers at Duke University successfully delivered CRISPR/Cas9 repressors to silence the Pcsk9 gene, which regulates cholesterol levels, in adult mice. The treatment resulted in reduced blood cholesterol levels and sustained gene repression for six months after a single treatment.
A novel technique integrates CRISPR technology with big data mining to identify functional roles of lncRNAs in chemotherapy resistance. The approach identified hundreds of new genetic units impacting sensitivity to chemotherapy, providing potential biomarkers for diagnosis and treatment.
A new gene therapy approach has been shown to reduce the need for blood transfusions in patients with severe beta-thalassemia. The treatment, known as LentiGlobin BB305 vector, was effective in eliminating the need for transfusions in 73% of patients, paving the way for potential treatment.
A new gene therapy has successfully treated transfusion-dependent thalassemia, a blood disorder that requires frequent red blood cell transfusions. The treatment produced positive outcomes in an interim analysis of two international clinical trials, with most patients becoming transfusion-free.
A gene therapy treatment for cerebral adrenoleukodystrophy (ALD), a fatal degenerative brain disease, has been successfully tested in a clinical trial. The treatment halted the progression of the disease in 15 out of 17 boys who participated in the trial, leading to minimal or no symptoms and normal lives.
A new liquid biopsy-based cancer model has revealed insights into the development of chemotherapy resistance in small-cell lung cancer. The study found that intratumoral heterogeneity, or differences in gene expression between tumor cells, contributes to rapid chemotherapy resistance.
Researchers developed a powerful mouse model using CRISPR-Cas9 and RCAS/TVA system to mimic glioma genetic alterations. The model enables the study of gene fusions and mutations in cancer progression and treatment response.
Researchers engineered a donor cornea with genes that inhibit vascularization, reducing the risk of tissue rejection in high-risk patients. The study shows promising results for gene therapy in preventing corneal graft rejection.
Researchers used machine learning to classify abnormal protein activity in tumors, identifying potential 'hidden responders' who may benefit from specific therapies. The study combined genetic data with machine learning approaches to predict response to inhibitors affecting cancer cells with overactive Ras signaling.
Scientists have developed a method to efficiently genetically modify T-cells from mice, allowing for targeted gene surgery and potential therapeutic applications. The technique involves using plasmids and CRISPR-Cas9 molecular scissors to introduce specific changes into the cells.
Researchers discovered a parasite-derived protein, H-IPSE, with potent therapeutic effects on chemotherapy-induced hemorrhagic cystitis in mice. The study found that H-IPSE alters host cell transcription, suggesting a novel approach to treating bladder pain and injury.
Researchers developed novel strategies to engineer more stable and active proteins, enhancing mRNA therapy potential. Sequence engineering improved protein expression, duration, and enzymatic activity in mice.
Researchers found that gene therapy using adeno-associated viral vectors can only temporarily alleviate congenital adrenal hyperplasia (CAH) by replacing the defective gene. A long-term solution requires targeting genetic mutations in adrenocortical stem cells, according to a study published in Human Gene Therapy.
Researchers used joint tissue biopsy to identify genes linked to disease progression and response to treatment in rheumatoid arthritis patients. This new technique holds promise for personalized therapy and improved patient outcomes.
Researchers have discovered nine new genes associated with osteoarthritis, a degenerative disease causing joint damage and pain. The findings could lead to the development of targeted therapies for this debilitating condition, affecting 10 million people in the UK.
A study by Hiroshima University researchers found that a specific gene group controls DNA damage response in hypoxic cancer cells, weakening the effectiveness of anticancer therapies. Suppressing this gene, DEC2, made cancer cells more sensitive to radiation treatment.
A team of researchers at Virginia Tech Carilion Research Institute improved the developmental skills of three girls with microcephaly, a genetic disorder affecting brain and body development. The girls made significant gains in skills and abilities after receiving intensive therapy for four hours a day for 10 days.
Researchers at UCLA have created a new method for targeted gene delivery using 'nanospears,' which can enable safer, faster and more cost-effective gene therapies. The nanostructures are biodegradable and can be mass-produced inexpensively, delivering genetic information with minimal impact on cell viability and metabolism.
New guidelines aim to improve care transitions for adults with DMD, emphasizing psychosocial issues, care coordination, and quality of life. Revised recommendations focus on preventing disease complications through prevention, earlier diagnosis, and better treatment.
A new study has identified 22 new genetic risk factors for stroke, providing extensive insight into the biology and pathways leading to the disease. The results demonstrate shared genetic influences with multiple related vascular conditions, including blood pressure and coronary artery disease.
A large international study has identified 22 new genetic risk factors for stroke, contributing to a better understanding of the disease's molecular mechanisms. The findings highlight the importance of genetics in identifying novel drug targets and may lead to personalized treatments for this complex disease.
A new study using recombinant adeno-associated virus (rAAV) delivery vectors found that ZMapp antibodies administered to mice resulted in 100% protection against Ebola infection. The study also compared different doses and routes of administration, with the best results achieved using a single rAAV-delivered antibody.
Northeastern researchers have discovered 36 new genes linked to cardiac disease, offering a promising direction for personalized medicine. The study used an innovative approach to analyze gene expression in mice and identified previously unknown genes that play a causal role in heart failure.
A bioengineering team has designed genetic 'circuits' using living cells to study microRNA expression. This research aims to improve synthetic biology and gene therapy by understanding how microRNAs function in different biological contexts.
Researchers at Kazan Federal University used gene and cell therapy to treat spinal cord injuries, improving motor functions and tissue structure. The approach, which uses umbilical cord blood cells to transport therapeutic genes, has potential applications for other demyelinating diseases like multiple sclerosis.
Researchers have developed a gene therapy that successfully treats a form of macular degeneration in dogs, which could lead to a human treatment for an untreatable disease. The therapy works by repairing the connection between retinal cells, restoring vision.
Researchers at Washington University in St. Louis have developed a method enabling effective insertion of large molecules into cells using acoustic shear poration and electrophoresis. The approach has achieved greater than 75 percent delivery efficiency of macromolecules, including DNA insertion, which is significantly more challenging.
Scientists have decoded the three-dimensional molecular structure of the healthy human huntingtin protein, enabling its functional analysis. This breakthrough could contribute to the development of new treatments for Huntington's disease.
Researchers found that disruption of ganglioside enzyme leads to cell overgrowth in Sandhoff disease organoids, mimicking large brains of patients. Gene therapy approach was successful in improving size and reducing ganglioside accumulation after treatment.