Articles tagged with Gene Therapy
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.
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.
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 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 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.
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.
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.
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 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.
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 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.
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.
Researchers have expanded the number of naturally occurring CRISPR-Cas systems, giving a wealth of potential new tools for large-scale gene editing. The discovery could lead to treating complex diseases associated with multiple genes.
A new Northwestern study reveals that brain motor neurons degenerate early in diseases like ALS, sending warning signals and showing defects. Targeting the brain's motor neurons could lead to long-term and effective treatment strategies.
Researchers genetically mapped the cell types of the mouse iris, revealing four new cell types and mapping genetic changes that occur when the iris dilates. This research may help connect genetic similarities between mice and humans, offering clues for developing new diagnostic tests and treatments for eye diseases.
A study published in eLife reveals that a small signalling protein called ARL4C is overexpressed in pancreatic cancer patients, facilitating their aggressive behavior. By inhibiting ARL4C, researchers have shown promise in reducing the spread of pancreatic cancer cells, opening up new therapeutic avenues.
A Charité study confirms the efficacy of gene therapy in improving muscle function and reducing mortality in children with spinal muscular atrophy. The treatment, onasemnogene abeparvoven, was found to be effective in children under two years of age, offering a promising alternative to existing treatments.
A recent study reveals that child maltreatment is associated with altered oxytocin gene methylation, leading to atypical brain structures and function. This finding suggests a potential biological mechanism for targeting during adolescence to improve the lives of victims of childhood abuse.
Researchers developed a novel model to identify specific genes and genetic alterations in multiple myeloma, stratifying the cancer's severity via DNA and RNA sequencing. This model revealed diverse subtypes and high-risk patients beyond current classifications.
Experts debate whether newborn genome sequencing should be routine, with some arguing it can save lives and be cost-effective. A phased rollout is advocated, with genomic information disclosed sequentially at appropriate ages. The rollout requires data quality improvement and informed consent.
A novel gene therapy has shown sustained expression of clotting factor VIII, leading to a reduction or complete elimination of bleeding events in patients with hemophilia A. The trial demonstrated improved production of coagulation factor VIII over prolonged periods.
Researchers from Genethon and Université de Paris have developed an AAV-based gene therapy that corrects skeletal damage caused by XLH in mouse models. The treatment targets liver hepatocytes to express therapeutic proteins, potentially offering a lifelong solution for this rare disease.
Researchers at the University of Kent identified strong evidence-based benefits of preimplantation genetic testing for aneuploidy (PGT-A) in IVF. Live birth rates per embryo transferred and per treatment cycle were significantly higher with PGT-A compared to regular IVF, especially in women over 40.
A genetic defect in the XIAP gene disrupts immune responses against bacteria, leading to an imbalance in the microbiome and promoting intestinal inflammation. The researchers discovered a potential therapeutic option by re-introducing antimicrobial molecules to mice with the genetic defect.
A new study investigates the effects of cord blood cell transplantation and curcumin administration on Tay-Sachs disease. The results show an increase in enzyme production and a decrease in inflammation after transplantation, as well as improved symptoms and reduced GM2 ganglioside levels when combined with curcumin.
Researchers at MIT and Harvard University have developed a way to selectively turn on gene therapies in target cells by detecting specific messenger RNA sequences. This technology can fine-tune gene therapies for applications ranging from regenerative medicine to cancer treatment, potentially reducing side effects and increasing efficacy.
Researchers at Duke University and UC Irvine identify Kenpaullone, a cancer drug, as an effective analgesic for chronic and challenging-to-treat pain. The compound enhances Kcc2 gene expression, which resets maladaptive genetic switches in neurons, leading to pain signal silencing.
Researchers from Kazan Federal University have developed a gene-cell preparation that uses membrane vesicles to target and kill cancer cells. The technology has shown promise in treating various types of cancer, including breast, lung, and colon cancer.
Researchers have identified eight genes in the Candida glabrata fungus that confer resistance to various drugs. The study allows for genetic testing to diagnose potential drug resistance, guiding treatment choices. The findings also highlight cross-resistance phenomena, where exposure to one drug can lead to resistance to another.
A gene therapy strategy has shown encouraging early results against Angelman syndrome, a neurodevelopmental disorder affecting approximately one in every 20,000 children. The therapy restores function of the UBE3A gene in neurons, preventing key signs of the condition and improving motor skill-learning and behaviors.
A novel computational platform called scAAVengr uses single-cell RNA sequencing to quickly evaluate viral vectors for delivering gene therapies to the retina with maximum efficiency and precision. This approach saves time and resources by identifying suitable candidates that can deliver therapy to affected parts of the retina accurately.
Researchers developed a new protein treatment that prevents glaucoma from forming in mice and reduces pressure in the eyes. The study provides new targets for therapies and aims to develop an injectable treatment for patients.
A group of researchers led by Indiana University School of Medicine's Benjamin Gaston will receive a research program project grant to fund the development of personalized therapeutic approaches for severe asthma. The grant will support three key projects focused on S-nitrosylation signaling, airway pH regulation, and androgen signaling.
A new study from the University of Pennsylvania School of Medicine found that the gene CDKL5 plays an essential role in the brain throughout life, even after childhood. Researchers discovered that reinstating CDKL5 activity in young adult mice with a genetic deficiency led to significant improvements in neurological problems.
Researchers report that 90% of patients who received gene therapy between 2009 and 2012 remain disease-free, with significant immune system differences observed among those treated. The therapy is most effective in younger children, but further work is needed to achieve high levels of gene correction in all patients.
Researchers conducted genomic evaluations of advanced malignancies to develop matched, individualized combination therapies. The study found that precision cancer therapy improved median overall survival rate by 3.9 months compared to standard care.
A study by Purdue University researchers has discovered a way to use gene therapy to turn glial brain cells into neurons, restoring visual function. This process is more efficient and less damaging than stem cell therapy, offering new hope for patients who have lost vision or motor skills after a stroke.