Articles tagged with Gene Therapy
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.
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.
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 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.
A special issue of Human Gene Therapy highlights Chinese research advancements in gene and cell therapy, including novel viral and nonviral vectors for gene delivery. The articles explore innovative applications of CRISPR technology in various disease areas.
Researchers at the German Primate Center have improved DNA transfer in gene therapy by modifying viruses to produce a protein called CD9, which increases exosome production and virus efficiency. This results in an 80% infection rate, making gene therapies more efficient.
A recent study revealed that patients with inherited DNA repair mutations in metastatic prostate cancer can derive similar benefits from treatment with standard therapies as other patients. Ongoing research explores the potential potency of targeted therapy with PARP inhibitors, which may offer additional therapeutic options for these ...
A new hybrid-capture sequencing assay has been established as the method of choice for detecting 'actionable' gene mutations in lymphoid cancer, with a high accuracy rate of 91%. The assay can be implemented by clinical laboratories into routine diagnostic workflows.
GlycoNet has awarded $3.5 million to researchers across Canada to explore glycomics and its impact on human health. The grants will support research into Parkinson's disease, Alzheimer's, drug-resistant fungal infections, and other areas.
A Tel Aviv University study proposes a novel approach to manipulate genes using a self-assembling platform that delivers nucleic acids to distinct cell subsets. The platform shows promise in treating autoimmune diseases and cancer by targeting specific cells and reducing inflammation.
Researchers have created a stealth virus that effectively recognizes and infects tumor cells using adapter molecules. The virus is protected by a novel protein shield that prevents immune system elimination, opening up avenues for treating aggressive cancers.
A global, multicenter study has found CAR T-cell therapy to be safe and effective in treating patients with relapsed or refractory B-cell ALL. The therapy achieved an overall remission rate of 81% within 3 months of treatment, with a median length of remission lasting up to 20 months.
Researchers found liver and neuronal toxicity with high doses of gene therapy delivered using an adeno-associated virus (AAV9) vector. The study warns about the importance of prioritizing patient welfare in gene therapy research.
Scientists at McGill University Health Centre and the University of Lorraine discovered a new cause of cblC, a rare condition affecting vitamin B12 processing. The study found epimutation, a gene modification causing silencing of one copy of the MMACHC gene, leading to severe health problems.
A new viral gene therapy delivers extra copies of the SOD2 and CTGF genes to protect healthy tissues from damage caused by radiotherapy. The treatment has shown promising results in preventing tissue damage and scarring in rats treated with radiotherapy, potentially improving breast reconstruction outcomes for women with breast cancer.
Researchers are exploring gene delivery strategies for osteoarthritis and rheumatoid arthritis, enabling sustained drug production at the site of disease. Clinical trials are underway in the U.S., with the first arthritis gene therapy recently approved in Korea.
A new study establishes a decisive link between immune cells and systemic sclerosis, providing a starting point for developing new therapies. Hundreds of macrophage genes are identified as crucial for the development of the disease.
Researchers at University of Wisconsin-Madison have developed an improved gene delivery tactic for treating glaucoma, a leading blinding disease. The new method doubles the transfer of genes entering target cells and spreads them more uniformly across the trabecular meshwork tissue.
A new study found that genetic variations in the CYP2C19 enzyme affect escitalopram levels, leading to improved therapeutic outcomes when doses are adjusted accordingly. The research identified a significant difference between patients with high and low enzyme expression, highlighting the potential for personalized medicine.
Recent advances in gene therapy have led to successful treatments for patients with serious medical conditions. Emerging genome editing technologies, such as CRISPR/Cas9, hold promise for broader and more effective gene therapy approaches.
Researchers have developed a new method to stabilize small interfering RNAs (siRNAs) by introducing phosphoramidate modifications, which enhances their stability and therapeutic potential. The study shows that the modified siRNAs maintain their gene silencing activity, making them suitable for various therapeutic applications.
Dr. Amelia Gallitano, a UA College of Medicine - Phoenix faculty member, has received a $175,000 grant to develop the first diagnostic test for schizophrenia. The test aims to determine whether an individual has schizophrenia or a specific subtype by leveraging genes activated in response to environmental events.
A gene therapy approach using AAV vectors reprograms alpha cells into functional insulin-producing beta cells, restoring normal blood glucose levels for an extended period. The strategy shows promise for treating autoimmune diabetes without immunosuppression.
Researchers identified shared immunity-related pathways involved in advanced stages of inherited blindness, hinting at potential therapeutic strategies. The study's findings also reveal similarities with common vision disorders like diabetic retinopathy and age-related macular degeneration.
A groundbreaking gene therapy trial has shown that over one year on from a single treatment, participants with haemophilia A are showing normal levels of the previously missing protein, effectively curing them. The results have particular significance as the first successful gene therapy trial for the haemophilia A.
Researchers announce innovative tools and therapies for hard-to-treat blood disorders, including gene editing platform CRISPR/Cas9. Interim results from a Phase I/II clinical study show restoration of all immune cell types in infants with X-linked severe combined immunodeficiency.
Researchers report remarkable benefits from new therapies for bleeding and clotting disorders, including emicizumab's high effectiveness in preventing bleeds in children with hemophilia A. Additionally, a gene therapy treatment provides durable improvement in clotting factor levels and long-term protection from bleeds.
Early evidence suggests that a gene therapy developed at St. Jude improves immune systems in infants as young as 2 months old with XSCID, offering broad protection and reducing the need for protective isolation. The treatment has been shown to be well-tolerated and effective in restoring immune function.
A team of researchers from the Broad Institute of MIT and Harvard has developed a new compound that can protect kidney cells from death and restore kidney function in multiple animal models of progressive kidney disease. The compound, called AC1903, works by blocking a damaging feedback loop involving the protein TRPC5.
The Phase 1/2 clinical trial showed that SPK-9001 reduced annualized bleeding rate by 97% and factor IX concentrate use by 99% in participants with hemophilia B. The data suggest a one-time infusion of SPK-9001 may sustain factor IX activity levels, reducing bleeding episodes.