Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
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.
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 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.
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.
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.
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 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.
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.
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.
Researchers at Salk Institute create a new version of CRISPR/Cas9 that can activate genes without creating DNA breaks, potentially treating diseases such as diabetes and muscular dystrophy. The technology operates epigenetically, influencing gene activity without changing the DNA sequence.
Researchers found a shift in cytokine profiles of mice with FVIII inhibitor development, including increased levels of pro-inflammatory cytokines. A negative correlation was also observed between risk factors for FVIII inhibitor development and age at gene therapy administration.
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.
A new gene therapy transplantation technique could improve treatment of neurodegenerative diseases by generating genetically engineered microglia-like cells in the brain. This technique has been tested on an experimental model for a metabolic disease and may have future therapeutic applications for other neurodegenerative diseases.
Scientists have made a breakthrough in treating neurodegenerative disorders and lysosomal storage diseases by transplanting hematopoietic stem cells directly into the brain, achieving therapeutic benefits faster than traditional methods. This innovative technique could pave the way for new treatments for Parkinson's, Alzheimer's, and o...
Researchers found seven genes with altered expression in melanoma patients, which could serve as biomarkers for diagnosis and therapeutic targets. The synthetic compound DM-1 induces cell death by increasing the expression of TOP-1 and ADK genes.
A new gene therapy has successfully produced sustained levels of clotting factor in adult hemophilia B patients, eliminating the need for regular infusions. The treatment, which uses a highly active variant of the clotting factor, achieved significant clinical benefits with no adverse effects.
The article examines the persistence of continuous electronic fetal monitoring as a standard of care, despite evidence that it increases risks. It also discusses the lack of mechanisms for ensuring medical professionals stay current with best practices and the role of malpractice law in setting boundaries on acceptable interventions.
Researchers designed new therapeutic DNA aptamers with diverse side chains to enhance interaction with targets. The study found that the hydrophobicity of side chains affects clearance from the bloodstream, providing a guide for designing better aptamers.
Researchers developed a novel gene therapy that lasts for over eight months, reducing methamphetamine in the brain and its stimulant effects. The therapy generates antibodies against meth, preventing it from reaching the brain.
A new gene therapy has shown significant improvements in vision for patients with Leber congenital amaurosis, a rare inherited retinal disease. The treatment, currently under FDA review, allows patients to navigate mazes and experience improved light sensitivity and peripheral vision.
A boy suffering from extensive skin damage due to epidermolysis bullosa was successfully treated with genetically modified stem cell transplants. The treatment resulted in the growth of high-quality, stress-resistant skin, allowing him to attend school and participate in social life.
A Phase 1 clinical trial has demonstrated encouraging data for gene replacement therapy in patients with spinal muscular atrophy type 1 (SMA1). The study found that high-dose gene therapy improved motor function and reduced the need for supportive care in patients treated with a single intravenous infusion.
A study published in Annals of Oncology has identified genetic markers that may predict resistance to a specific therapy used for treating advanced prostate cancer. The researchers found that understanding the genetic markers of tumors can help healthcare providers match a therapy that is likely to succeed on the first try.
Researchers at Case Western Reserve University School of Medicine have developed a gene therapy that can prevent the progression of hearing loss and preserve hearing in people with Usher syndrome type III, a form of hereditary hearing loss linked to defects in sensory hair cells.
German researchers have made significant advancements in human gene therapy, including virotherapy capable of destroying tumor cells and engineered hematopoietic stem cell delivery systems. These innovations hold promise for treating immunodeficiencies and genetic diseases.
A groundbreaking study has found that injecting DNA into injured horse tendons and ligaments can cure lameness within two to three weeks. The gene therapy technology also showed no adverse side effects, with the horses returning to full health after just two months.
Researchers have identified 13 new genetic risk variants for restless legs syndrome, a condition affecting up to one in ten people of European ancestry. The findings suggest that the condition may be related to neurodevelopmental disorders and offer clues for developing new treatments, including drugs like thalidomide.
A group of inflammatory proteins called 'IL-36' play a significant role in the development of psoriasis and can be blocked without posing a risk to patients. The findings open up the possibility for new drugs to tackle this debilitating condition.
M344 altered key gene expression in a cellular model of AD, preventing cognitive decline and improving memory. In mouse models, low-dose administration of M344 prevented cognitive decline and improved learning and memory.
A new study successfully uses a shortened version of the CEP290 gene to treat Leber congenital amaurosis type 10, a rare genetic disorder causing blindness. The treatment shows significant improvement in photoreceptor survival and function.
A recent study has identified 150 genetic drivers of diffuse large B cell lymphoma, a common form of blood cancer. The research, led by Duke Cancer Institute scientists, found correlations between specific genes and treatment responses, opening up new avenues for targeted therapies.
A new study in The FASEB Journal reveals that over-expression of the GTRAP3-18 gene is linked to obesity in humans. Inhibiting its interaction with POMC might be a strategy for treating leptin/insulin resistance in patients with obesity and type 2 diabetes.
Researchers report that a gene therapy to treat cerebral adrenoleukodystrophy has effectively stabilized the disease's progression in 88 percent of patients. Fifteen out of 17 patients had stable neurologic functioning more than two years after receiving the treatment.
A Swedish national adoption study found that children of divorced parents are more likely to get divorced due to genetic factors, not environmental influences. The study suggests that targeting basic personality traits linked to divorce may be a better approach for therapists.
Researchers used gene therapy to express melanopsin in mouse retinas, restoring visual function and improving pupil constriction. The treatment was effective for up to 15 months, showing enhanced visual responses and light avoidance behaviors.
A new study found that customizing warfarin dosing to a patient's genetic and clinical profile reduces adverse outcomes compared to clinically-guided dosing. The study involved 1,650 patients undergoing hip or knee replacement, who were randomly assigned to either genotype-guided or clinically-guided dosing.
Researchers used gene therapy to improve heart function in a large animal model, finding a 25% improvement in left ventricular function and a 20% improvement in left atrial function. The treatment also reduced heart size by 10%.
Researchers developed a novel gene delivery approach using scaffold/matrix attachment region (S/MAR) to enhance long-term transgene expression in proliferating cells. The modified vectors were able to establish colonies and maintain stable transgene expression in HeLa cells.
Researchers at Institut Pasteur successfully restored hearing and balance in a mouse model of USH1G syndrome using gene therapy, offering new hope for the treatment of hereditary deafness. The study demonstrates that inner ear defects caused by mutations in the USH1G gene can be reversed even after birth with durable efficacy.
Researchers used gene therapy to stop the immune response that causes multiple sclerosis in mouse models, producing near-complete remission. The treatment combined a brain-protein gene with an existing medication, showing significant potential for treating multiple sclerosis and other autoimmune disorders.
A potent gene immunotherapy strategy induces robust immune tolerance in mice with multiple sclerosis, reversing symptoms and stabilizing brain function. The treatment, using a viral vector to deliver myelin sheath protein, protects against disease progression and restores mobility.
A special issue in the Journal of Dental Research explores recent scientific and technical advances in craniofacial development and genetics. The discoveries reported could improve diagnosis and treatment of challenging craniofacial and dental anomalies.
Researchers identified two cytokines and genetic markers associated with progressive MS, which may enable the development of a treatment to prevent disease progression. A simple genetic test could identify patients at risk, and a medication is already in development.
Scientists at Oregon State University developed a new assay to study the otoferlin protein, essential for hearing. They found a truncated version of otoferlin that can function in sound encoding and validated a method for characterizing large membrane proteins.