Articles tagged with Gene Therapy
A gene called Fer2 has been identified as a key player in protecting dopaminergic neurons from degeneration. In flies and mice, overproduction of Fer2 reduces oxidative stress and prevents neurological defects characteristic of Parkinson's disease.
A clinical trial found that genomic sequencing enabled 107 patients to receive matched therapy, increasing treatment options for those experiencing cancer relapse. The study identified previously unknown mutations and showed promise for using circulating tumor DNA to identify targetable alterations.
Scientists have developed a genetic screening platform to identify genes that enhance immune cells' persistence and ability to eradicate tumor cells. By combining these genes with existing CAR-T cell therapy, researchers were able to engineer T cells that are more effective at eliminating tumor cells.
A novel surgical approach and synthetic viral vector Anc80L65 successfully delivered genetic cargo to the inner ear of nonhuman primates, paving the way for a potential treatment for hearing loss and vestibular disorders. The study's findings suggest that this method could be translated to humans.
Researchers have introduced a new mRNA delivery method to treat muscular dystrophy, which has already proven successful in mice. The method uses messenger RNA to correct genetic defects and has shown promise in clinical trials.
Researchers have genetically engineered a rat model of Down syndrome to test new therapies and explore the condition's unique genetics. The rats exhibit cognitive impairments, anxiety, and hyperactivity similar to humans with Down syndrome, providing a valuable tool for medical research.
Researchers discovered that an inorganic polyphosphate released by nerve cells contributes to the death of motor neurons in people with ALS and frontotemporal dementia. The study found that lowering levels of this toxin may be an innovative therapeutic strategy for diverse types of ALS/FTD.
Scientists developed a powerful new tool called Giraffe to improve genomic research by leveraging global genetic diversity. The tool allows for the use of a diverse pangenome reference point, enabling faster and more sensitive comparisons of short-read human genome sequences.
A clinical trial at UC Davis Health showed that cellular therapy offers promise for patients with late-stage Duchenne muscular dystrophy, stopping deterioration of upper limb and heart functions. The therapy appears to be safe and effective in improving skeletal muscle and cardiac function.
A new mathematical framework has been created to study fitness landscapes of regulatory DNA, enabling the prediction of gene expression changes. The framework uses a neural network model trained on millions of experimental measurements to decipher the evolutionary past and future of non-coding sequences.
Researchers uncover the pleiotropic functions of hnRNPK in regulating skeletal muscle cell differentiation, including inhibition of myoblast differentiation and suppression of genes involved in endoplasmic reticulum stress. The study suggests that targeting hnRNPK could be a potential therapeutic strategy for treating human disorders.
Researchers have discovered a genetic difference in the meniscus that makes about 50% of females more prone to developing knee osteoarthritis than males or other females. The study suggests potential for a blood test to identify high-risk individuals, allowing for early interventions and prevention strategies.
Scientists at the University of Texas at Austin have redesigned a key component of the widely used CRISPR-based gene-editing tool Cas9 to be thousands of times less likely to target the wrong stretch of DNA. The new version, called SuperFi-Cas9, is as efficient as the original but reduces off-target interactions, making it potentially ...
Researchers discover a genetic mechanism linking ALS and dementia to UNC13A protein corruption, providing hope for new treatments by blocking corrupted instructions. The study found that genetic variants increase the risk of UNC13A mRNA corruption in patients with ALS and FTD.
A Phase 3 clinical trial shows that gene therapy can end the need for monthly blood transfusions in children with transfusion-dependent thalassemia. The treatment uses a patient's own stem cells modified with a healthy hemoglobin gene, allowing patients to achieve transfusion-free status within months.
The five-year grant will be used to mentor junior scientists, leveraging City of Hope's expertise in cancer treatment and patient care. The program will provide students with hands-on experience in cell-based therapies, regulatory approval, and commercialization.
Scientists have developed a new therapy called CINDELA, which employs CRISPR-Cas9 to kill cancer cells while leaving normal tissues intact. The treatment targets specific mutations found in cancer cells and induces cell death through DNA double-strand breaks.
Researchers discovered how pre-transplant chemotherapy facilitates replacement of brain's innate immune cells by transplanted stem cells. This process involves microglial cell death and senescence, but is not harmful to the brain, as it is quickly replaced by bone marrow-derived macrophages.
Research by Shan Jiang found that introducing nature views into hospital corridors can significantly ease confusion and anger in navigating large medical complexes. The study revealed that participants used shorter time and walked less distance to complete wayfinding tasks when exposed to green spaces.
Researchers from Trinity College Dublin have pinpointed a key driver gene, SARM1, that contributes to impaired vision and blindness. Deleting this gene shows promise in preserving vision, suggesting targeted therapies may offer long-lasting preservation of sight for various ocular conditions.
Researchers identified a novel mutation in 9% of relapsed pediatric AML cases, suggesting a new subtype of the disease. The UBTF tandem duplication is associated with poor survival rates and higher likelihood of minimal residual disease positivity.
A new study has identified the critical genes most likely to cause coronary heart disease and trigger heart attacks. The research provides a prioritized list of 162 genes, which will enable more accurate genetic testing and targeted therapies for patients at risk of coronary heart disease.
A recent study found that gene therapy delivery vectors were unlikely to cause the blood malignancies reported in trials; however, the exact cause remains a mystery. The study's results suggest that cancer risk in sickle cell disease may be more complex than initially thought, warranting further investigation.
Researchers at Eötvös Loránd University have identified the molecular mechanism behind an important form of RNA modification, which can lead to genetic disorders. The discovery could pave the way for targeted RNA modifications and gene therapies.
Researchers at Mayo Clinic Cancer Center developed a machine learning algorithm that integrates genetic data from over 5,000 patients to predict patient benefit from chemotherapy and immunotherapy. A 32-gene molecular signature was identified, providing prognostic information and predicting patient response to immunotherapy.
Researchers developed a new RNA editing technology that makes efficient use of native ADAR enzymes to correct disease-causing mutations in RNA. The technology holds promise as a gene therapy for treating genetic diseases like Hurler syndrome and cystic fibrosis, with promising results in mouse models.
A team of researchers has identified over 250 gene activators in human cells, expanding our understanding of transcriptional regulation and its role in cancer. The study also reveals new insights into how proteins interact with each other to regulate gene expression, potentially leading to the development of targeted therapies.
Pulmonary lymphangioleiomyomatosis (LAM) is a rare cancer affecting up to 1 in 1 million women worldwide, characterized by uncontrolled tumor cell growth. Researchers aim to identify new therapeutic targets using extracellular vesicles, with the goal of developing new therapies for LAM patients.
Researchers at Johns Hopkins Medicine found that certain stem cells have built-in tracers made of sugars that can track their movement in living tissues. The discovery could streamline and advance restorative research for diseases of the brain.
A recent study published in Developmental Cell reveals that Kras mutation causes chromatin rearrangement, leading to stem-like cell regeneration and tumor onset. The team discovered a protein complex called AP-1 as the mediator of this process, which can be targeted with small-molecule drugs.
Biomedical engineers at Duke University have developed a gene therapy that helps heart muscle cells electrically activate in live mice. The approach features engineered bacterial genes that code for sodium ion channels, which could lead to therapies to treat electrical heart diseases and disorders.
Researchers at Rosalind Franklin University have identified a new therapeutic approach for treating cystic fibrosis. The treatment uses antisense oligonucleotides to restore CFTR function by removing stop mutations. This strategy has shown promise in treating CF patients with class I mutations and similar types of mutations.
A new generation of cholesterol-lowering drugs is being developed to target the PCSK9 protein, which regulates LDL receptor degradation on cells. These therapies have shown promise in reducing LDL levels with fewer side effects than statins.
Researchers found that dexamethasone administration after gene therapy increased liver transgene expression in mice. This finding suggests a potential strategy for reducing steroid side effects in future clinical trials.
Researchers at Cold Spring Harbor Laboratory develop a novel method to modify the CFTR gene, allowing for the production of functional protein in patients with certain mutations. The technique involves using antisense oligonucleotides to skip over the mutation and produce a partially functional protein.
A novel gene therapy approach using an adeno-associated virus (AAV) has shown promising results in treating Huntington's disease. The therapy targets the mutated huntingtin gene (HTT), providing efficient and precise processing of a primary artificial microRNA, leading to potent pharmacological activity for HTT lowering.
A team of researchers at Harvard's Wyss Institute and ETH Zurich have developed a computational approach to identify genomic safe harbors (GSHs) with high potential for safe insertion of therapeutic genes. The study validated two GSH sites in adoptive T cell therapies and in vivo gene therapies for skin diseases.
Researchers from Osaka University created patient-derived heart cells that exhibit reduced contractility and impaired desmosome assembly when carrying a mutation associated with arrhythmogenic cardiomyopathy. Replacing the mutated gene restored normal function, suggesting a potential treatment approach for this disease.
Researchers developed a new drug delivery system using engineered DNA-free virus-like particles (eVLPs) to package and deliver therapeutic levels of gene-editing proteins. eVLPs enabled safer in vivo delivery with comparable or higher efficiencies, overcoming multiple structural limits to potency.
Researchers found that glioma cells with mutated ATRX have reduced Chk1 activity, leading to dysregulated cell cycle and heightened sensitivity to ATM inhibitors. The study suggests that combining radiation therapy with these inhibitors may improve treatment outcomes for patients with this gene mutation.
A recent study published in Science Translational Medicine identified a novel causative gene, BAG5, for dilated cardiomyopathy, a leading cause of heart failure. The researchers found that mutations in this gene can lead to cardiomyopathy and found a potential treatment alternative using adeno-associated viruses (AAV) gene therapy.
Researchers developed a gene therapy called Targeted Augmentation of Nuclear Gene Output (TANGO), which boosts SCN1A protein production in brain cells. The treatment restored normal cell function and reduced seizures in lab mice with Dravet syndrome, offering hope for the first direct treatment of the fundamental cause.
A new machine learning model, RefMap, has identified 690 genetic risk factors for motor neurone disease, a five-fold increase from previous estimates. This discovery could lead to the development of new treatments and personalized medicine for patients with MND.
Researchers successfully engineered mesenchymal stromal cells to carry and deliver therapeutics specifically to targeted tissues, offering a precise and reliable approach for treating diseases. This novel cargo-carrier, dubbed 'Cargocytes,' retains most of its cellular functionality while greatly enhancing therapeutic capacity.
Researchers at WVU are studying the Musashi proteins to understand their role in retinal degeneration and develop a universal therapy. By investigating protein translation and gene suppression, they hope to identify potential pathways to boost protein production and slow vision loss.
Researchers developed a color-coded test that quickly signals whether medical nanoparticles deliver their cargo into target cells. The tool, tested in mouse cells and living mice, assesses nanoparticle formulations on their ability to escape cellular defenses and reach the cell's interior.
Researchers developed a non-muscle targeted gene therapy that enhances muscle fiber repair and improves muscle function in LGMD 2B patients. The treatment, administered via a single injection, reduces muscle degeneration and restores myofiber size and muscle strength.
A new study reveals the sophisticated mechanism by which adenoviruses infect human cells and transfer foreign DNA into their nucleus. Protein V plays a crucial role in increasing the virus particle's stability and preventing premature DNA release, which triggers an anti-viral alarm system.
Researchers found that a Parkinson's disease mutation mislocalizes iron in activated microglia, leading to toxic iron accumulation. This mislocalization may explain the disease's progression and provide a basis for therapies targeting LRRK2.
A novel gene delivery technique has successfully restored hearing in mice with a mutated STRC gene, which is the second most common genetic cause of hearing loss. The approach could be used to deliver other large genes and may provide a broad window of opportunity for treatment from babies to adults.
Researchers are exploring how an engineered adeno-associated virus (AAV) can compensate for missing protein or swap out genetic mutations that cause vision problems. AAV has been found to be beneficial and is being used as a tool to deliver genes that work as they should.
The Nixon Visions Foundation has given a significant gift to support studies of the PRPH2 gene linked to macular dystrophy and boost stem cell research aimed at developing early diagnosis and a cure for this devastating genetic eye disease. Researchers hope to make a tremendous impact on people with this inherited eye disease.
A study published in Allergy reveals the importance of PU.1 transcription factor in regulating CCL17 gene expression, which contributes to allergic diseases. The research found that suppressing PU.1 can reduce inflammation in asthmatic mice, paving the way for novel treatments.
A new gene therapy called LentiGlobin has been shown to completely eliminate episodes of severe pain caused by sickle cell disease and restore blood cells to their normal shape. The treatment uses a patient's own stem cells and has the potential to give people with this disease their life back.
Researchers have developed Find Cut-and-Transfer (FiCAT) technology, a tool capable of accurately writing small and large genes. FiCAT allows precise insertion of large fragments into the genome, enabling development of therapeutic solutions for diseases like Duchenne muscular dystrophy and hereditary blindness.
Researchers at Georgia Institute of Technology have identified a key class of genetic changes associated with cancer, which may be missed by current gene expression analyses. These 'hub genes' play a central role in shaping the network structure of cancer cells and could serve as new targets for targeted gene therapy.
A new gene and stem-cell therapy has been proven to be effective in treating Epidermolysis Bullosa (EB), a genetic skin disorder, without any side effects. The treatment, which involves transplanting genetically modified skin cells, has resulted in stable results after five years, with the patient now 13 years old.
A NIH study has identified a molecular link between a gene mutation and late-onset retinal degeneration, a rare eye disease. The researchers found that the diabetes drug metformin and gene therapy may be effective treatments for the condition, which can cause abnormal blood vessel growth and deposits of apolipoprotein E.
The study used multigene sequencing to identify genomic alterations in patients with metastatic breast cancer. Patients with genomic alterations ranked as ESCAT I/II saw improved progression-free survival with targeted therapies matched to their genomic changes, while those without these alterations did not benefit from the treatment.
A recent study by NTU Singapore and Singapore General Hospital found that mutations in the DDX3X gene are responsible for chemotherapy resistance in some blood cancer patients. The study also discovered that STAT inhibitors can effectively kill lymphoma cells with DDX3X mutations, providing hope for new treatment options.