Kazan researchers compared direct gene and blood cell-mediated therapy for spinal cord injuries, revealing similar recovery outcomes in rats. The study found that both therapies preserved myelinated fibers, with cell-based therapy extending therapeutic influence over longer distances.
Researchers develop novel mouse model for Leber hereditary optic neuropathy and demonstrate successful gene therapy in improving visual function. The approach could aid therapy development for various mitochondrial diseases, with a clinical trial currently underway.
A new gene therapy approach delivers the ITGA7 gene to mice with Duchenne muscular dystrophy, reducing symptoms and extending life span by over 10 weeks. The method overcomes previous immunological issues, making it a promising novel treatment for DMD.
Scientists have successfully used gene therapy to fully restore vision in a mouse model of Leber congenital amaurosis-1, a genetic disorder causing severe visual impairment. The treatment, which replaced the deficient retGC1 protein, showed long-lasting results and supports clinical testing for human patients.
A review article highlights barriers to overcome in CAR T cell therapy for lymphoma, including physical barriers and immunosuppression. The study explores factors related to tumor biology and immunology compared to treatment response in patients with lymphoma.
Harvard researchers have reconstructed an ancient virus effective at delivering gene therapies to liver, muscle, and retina. The discovery may lead to novel vectors for safer and more potent gene therapies.
Patients on triple therapy had the same rates of adverse cardiac events as dual therapy but experienced more complications and bleeding; researchers recommend reevaluating treatment regimens for this population.
Researchers reconstructed an ancient virus to deliver gene therapies, targeting liver, muscle, and retina. The discovery could lead to safer and more potent treatments for various diseases.
Scientists found that additional genes can compensate for knocked-out genes and mitigate consequences. In a study on zebrafish, researchers identified emilin 3B as a rescuing gene for the egfl7 gene, which regulates blood vessel growth.
The CRISPR/Cas system has revolutionized biological research, enabling targeted DNA changes in living cells. This innovation is now being applied to clinical medicine, promising significant advancements in drug therapies, agriculture, and food products.
The National Academies have launched an international initiative to address the technical, social, and ethical issues surrounding human genome editing. The initiative aims to provide a comprehensive understanding of human genome editing and its implications for improving human health and boosting food production.
Researchers at Harvard University and UC San Diego have developed a new software that predicts the most active guide RNAs for specific gene targets, facilitating faster and more efficient genome engineering experiments. This breakthrough has the potential to accelerate discoveries in gene therapies and basic genetics research.
Researchers found that vision-restoring gene therapy leads to strengthening of visual pathways in the brain, even after decades of near blindness. The treatment restored nearly intact retina-brain connections, suggesting that unused pathways rebuilt themselves.
Researchers at Boston Children's Hospital and Harvard Medical School used gene therapy to restore hearing in mice with genetic deafness. The treatment successfully restored the ability of sensory hair cells to respond to sound and partially restored actual hearing in deaf mice.
A UK trial has shown significant benefits in lung function for patients with cystic fibrosis who received gene therapy. The treatment involves replacing the faulty gene responsible for the disease and was found to be safe and effective. Further research is needed to improve its effectiveness before it can be used as a clinical treatment.
A phase 2 trial has shown that gene therapy can improve lung function in people with cystic fibrosis, with a significant benefit seen in patients who received the treatment. The technique uses inhaled molecules of DNA to deliver a normal working copy of the CFTR gene to lung cells.
A clinical trial is underway at the Royal Alexandra Hospital to test a new gene therapy treatment for choroideremia, a genetic disorder that causes inherited blindness. The treatment involves injecting a customized virus into the retina to transfer a healthy gene, with promising results already seen in early patients.
Researchers have identified a gene, PRDM8, linked to early loss of night vision in animal models. Gene therapy for Leber's congenital amaurosis shows promising results, but temporary improvements are seen.
Dr. Qizhen Shi's team aims to investigate a novel gene therapy approach that induces immune tolerance for hemophilia A, overcoming the challenge of inhibitory antibodies. The project seeks to develop a clinically translatable gene therapy protocol that provides therapeutic FVIII protein.
A nationwide survey shows a significant growth in pediatric proton therapy patients, with 56% under age 10. Proton therapy offers precise treatment for various tumor diagnoses, including ependymoma and medulloblastoma.
Dr. Ana Buj Bello, a leading expert in gene therapy, has received the Outstanding New Investigator Award for her groundbreaking research on treating myotubular myopathy through gene therapy. Her work has shown promising results in animal models and paves the way for a potential clinical trial to treat children with this rare disease.
A new study by Moffitt researchers found that men treated with androgen deprivation therapy for prostate cancer have a higher risk of cognitive impairment. The study also identified a possible genetic link between the GNB3 gene and cognitive problems during ADT treatment.
Researchers developed a unique approach using microscopic gas bubbles to deliver directly to cancer cells a viral gene therapy in combination with an experimental drug. The treatment strategy used a novel 'cancer terminator virus' and demonstrated enhanced prostate cancer cell death while sparing healthy cells in preclinical experiments.
Researchers successfully restored bilirubin levels to those found in healthy animals using gene therapy, offering new hope for the treatment of Crigler-Najjar syndrome. The therapy involved transferring a copy of the UGT1A1 gene into liver cells using an AAV vector, resulting in long-term correction without immunosuppression.
A closer look at the DNA surrounding the Huntington's disease (HD) gene reveals critical regions controlling its expression. Changes in these regions can delay or accelerate the disease, with some individuals receiving protection from the mutant gene.
Gene therapy for Leber congenital amaurosis improves vision and retina sensitivity within weeks of treatment, with peak benefits after one to three years. However, these gains diminish over six years, revealing the need for further research to sustain restored vision.
Researchers at Icahn School of Medicine at Mount Sinai have developed a gene therapy approach that targets and corrects genetic mutations linked to heart failure. The treatment uses molecular scissors to cut out diseased genes and replace them with normal ones, restoring healthy cardiac cell function.
A study published in JAMA found that gene therapy resulted in clinical improvement for children with Wiskott-Aldrich syndrome, a rare immunodeficiency disorder. The treatment, which involved infusing corrected stem cells back into patients, led to the resolution of severe eczema and recurring infections.
Randomized clinical trials demonstrate endovascular therapy is highly beneficial in treating acute ischemic strokes. The new studies, published simultaneously in NEJM, attribute the improved results to superior surgical technology and improved neuroimaging criteria.
A new study comparing gene therapy to half-matched transplants for SCID-X1 reveals that gene therapy leads to faster immune development, fewer infections, and reduced hospitalizations. Gene therapy shows promise as a viable alternative treatment for infants with this rare immune disorder.
A study published in BMC Health Services Research found that early physical therapy following an initial episode of acute low back pain resulted in lower healthcare costs and reduced use of resources. Patients who received physical therapy within 14 days had significantly lower costs for advanced imaging, injections, surgery, and opioids.
A new study finds that carotid baroreflex activation therapy (BAT) improves NYHA Class, quality of life scores and exercise capacity in symptomatic advanced heart failure patients receiving optimum drug therapy. BAT was safely implanted with few complications and no adverse effects.
Researchers at UC San Diego have identified a gene variant in the SORL1 gene that may be used to predict people most likely to respond to an investigational therapy for Alzheimer's disease. The study found that variants of the gene confer some protection from AD and are associated with reduced beta amyloid peptide production in neurons.
A recent study has found that mutations in the APC2 gene cause Sotos-like symptoms, including nervous system-related issues and abnormal brain structure. The research team also discovered that APC2 is a crucial downstream gene of the NSD1 gene, which is responsible for Sotos syndrome.
Researchers at UT Dallas have created a novel gene-delivery system that shuts down after delivering a gene, offering a potential new strategy for treating diseases. The approach sidesteps health problems associated with permanent gene alteration.
Researchers have developed a new algorithm called Gene Rank (GR) to describe gene connectivity, which can be used for disease prognosis and early cancer detection. GR is based on gene expression data and reflects how well a particular gene is connected to other genes.
Pioneers in gene transfer technology, Drs. Mulligan and Miller developed the first DNA-based vectors to stably transfect cells with selectable markers. Their work laid the foundation for retroviral vector-based gene therapy strategies, leading to clinical testing and potential treatment of genetic diseases.
Researchers at Johns Hopkins Medicine have developed a new nanoparticle-based gene therapy that effectively kills brain cancer cells in rats and lengthens their survival. The treatment uses biodegradable nanoparticles filled with genes for an enzyme that turns a compound into a potent killer of cancer cells.
A study found that AAV vector integration and microRNA expression influenced liver cancer development after gene therapy. The study highlights the importance of considering vector design features when designing AAV vectors for gene therapy.
Researchers have identified a critical safety parameter related to AAV gene therapy, which can increase liver cancer risk. They found that modifying the vector to avoid targeting specific genes reduces cancer incidence, paving the way for safer gene therapies.
A new study found that adding radiation treatment to hormone therapy saves more lives among older men with locally advanced prostate cancer, reducing cancer deaths by nearly 50 percent. The treatment was also associated with fewer deaths from any cause.
Katherine High, Amit Nathwani, Arthur Nienhuis, and Andrew Davidoff honored for their groundbreaking research on gene therapy for hemophilia B. They developed successful clinical applications using adeno-associated virus vectors and demonstrated stable therapeutic expression of the clotting factor.
Researchers developed a gene therapy that boosts Factor IX levels in men with severe hemophilia B, resulting in significant reductions of 90-94% in spontaneous bleeding episodes. The treatment has enabled patients to adopt more active lifestyles without needing frequent clotting factor injections.
Researchers found that people with one inactive copy of the NPC1L1 gene had lower LDL cholesterol levels and a 50% reduced risk of coronary heart disease. The study suggests that targeting this gene may provide protection against heart attack, but further research is needed to confirm its effectiveness.
A study published in the Journal of Creativity in Mental Health found that animal-assisted therapy significantly reduced anxiety and loneliness symptoms among college students. The program, led by Dr. Leslie Stewart, involved interacting with a registered therapy dog named Sophie, resulting in a 60% decrease in self-reported symptoms.
Researchers developed an AAV-mediated gene therapy approach to correct hyperbilirubinemia in a mouse model of Crigler-Najjar syndrome type 1. The treatment achieved significant, long-lasting reductions in bilirubin levels, with 50-70% reductions maintained throughout the study.
A new form of gene therapy for SCID-X1 appears effective and safe, correcting the disease with a functioning immune system in seven out of eight patients. The therapy's long-term safety is still being monitored, but preliminary results suggest a reduced risk of leukemia compared to previous trials.
A modified y-retrovirus vector has been found to restore the immune systems of children with X-linked severe combined immunodeficiency, a rare and life-threatening inherited condition. The new approach is equally effective at restoring immunity and may be safer than previous gene therapy methods.
A new study suggests that combination treatment may not be necessary for all patient populations with the disease. Patients over 65 years old with small tumors and low risk tumor sizes achieved comparable survival after treatment with adjuvant radiation therapy alone, without undergoing endocrine therapy.
A Penn researcher and CVS Health physician suggest an alternative payment model for gene therapy, which would replace single large payments with annuity payments over a defined period. This approach could help ensure sufficient investments in expensive gene therapies by spreading out payments and linking them to evidence of continued e...
The Pioneer Award recipients have made significant contributions to gene therapy for retinal degeneration. Their research has led to proof-of-concept studies demonstrating the feasibility of using gene therapy to repair photoreceptor defects in the eye.
Researchers at the University of Pennsylvania have successfully treated lysosomal storage disease MPS I using gene therapy, reducing harmful protein accumulation and improving symptoms. The treatment involves direct gene transfer into glial and neuronal cells in the brain and spinal cord.
A new study found that mindfulness-based cognitive therapy significantly reduced non-mental health care visits among patients who were high health service users. Over eight years, this translates to a reduction of nearly 2,500 visits to primary care physicians, emergency departments, or non-psychiatric specialists in Ontario.
Researchers at the University of Missouri School of Medicine have developed a gene therapy that protects mice from a life-threatening heart condition caused by muscular dystrophy. The therapy targets a different gene involved in the heart's response to stress and shows significant improvements in overall heart health.
A new clinical trial has been initiated to assess gene therapy for patients with heart pumps, with the goal of improving heart function. The trial will evaluate the effectiveness of a gene therapy that increases SERCA2a protein levels in heart muscle cells, providing potential relief from advanced heart failure.
Dr. Brenner's work has contributed significantly to advancing the field of gene transfer using retroviral vectors in cancer immunotherapy. He is recognized for his contributions to developing genetically modified T cells that can effectively target tumors.
Researchers have developed a promising new approach to editing gene transcripts, which uses targeted oligonucleotide drugs. This technique has already shown promise in treating diseases such as Duchenne Muscular Dystrophy and spinal muscular atrophy.
Researchers developed a new genome editing method, TALEN-HDAdV, which increases gene-correction efficiency in human induced pluripotent stem cells. This technology has the potential to model human diseases and develop cell replacement therapy, offering hope for treating genetic diseases.
Columbia University researchers create a way to develop personalized gene therapies for patients with retinitis pigmentosa, a leading cause of vision loss. They use induced pluripotent stem cell technology to transform skin cells into retinal cells, which are then used as patient-specific models for disease study and preclinical testing.
Researchers found that patients treated with proton therapy had significantly higher disease-free survival rates (72% vs. 50%) compared to those receiving intensity modulated radiation therapy (IMRT). Tumor control also improved at the longest follow-up for proton therapy-treated patients (81% vs. 64%).