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.
Dr. Christof von Kalle has received a Pioneer Award from Human Gene Therapy for his leadership and accomplishments in the field of cell and gene therapy. He is recognized for his seminal contribution to vector integration, a critical feature of retro- and lentivirus-based vectors.
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%).
Dr. Frederic D. Bushman is recognized for his pioneering work on HIV reproduction and its application to advancing gene delivery methods. His research has contributed to the development of new vectors and targeting methods, informing safety profiles.
A recent study published in Nature Communications discovered a correlation between the number of biological functions a gene has and its response to environmental changes. The research found that genes with more biological functions exhibit less protein expression change in response to temperature, challenging the long-held assumption ...
Researchers successfully delivered a replacement SMN1 gene to animal models of SMA, extending their survival. The study demonstrates that enough copies of the SMN1 gene can be delivered to motor neurons in the spinal cord.
A novel RNAi therapy successfully blocks production of the dysfunctional huntingtin protein, causing Huntington's disease. The treatment reduces mutant Htt levels and disease symptoms in a mouse model without causing neurotoxicity.
Dr. James M. Wilson has dedicated his research to developing gene therapy and vectors for treating inherited diseases. He has made seminal contributions to adenoviral and AAV vector technologies, enabling the successful development of commercial products across various disease targets.
Researchers investigated four trained therapy dogs and found no stress during group therapy. The study established that freedom relaxes the dogs, reducing cortisol levels, and symptoms of stressed dogs include hair loss and anxiety-like behaviors. Regular supervision is recommended for therapy dogs to ensure their well-being.
Researchers at Ohio State University found that microRNA-3151 promotes AML-cell growth and survival by blocking tumor-suppressor gene TP53. High expression of miR-3151 is associated with a bad prognosis, while inhibition with the drug bortezomib offers a possible therapy.
A new gene therapy has shown promising results in improving hind limb function in rats with spinal cord injuries. The treatment involves delivering a scar-busting gene that promotes the survival of nerve cells and reduces inflammation, leading to improved mobility and tissue repair.
Marina Cavazzana and Adrian Thrasher have been honored with the Pioneer Award for basic and clinical gene therapy for immunodeficiency disorders. They are pioneers in treating life-threatening inherited diseases of the immune system with gene therapy, using a patient's own modified stem cells.
Researchers used a rat genomic gene-chip to profile hippocampal gene expression changes after electroacupuncture therapy. The results showed that electroacupuncture regulates the expression of specific genes involved in depression, including Vgf and Igf2. These findings suggest that electroacupuncture may be a useful treatment for depr...
Genethon, a non-profit organization, has partnered with Esteve to manufacture the gene therapy AAV9-hsulfamidase for treating Sanfilippo syndrome. This agreement supports clinical development and advances the treatment towards trials.
A new gene therapy approach has been shown to be safe and well-tolerated in a clinical trial of four children with mucopolysaccharidosis type IIIA, an inherited lysosomal storage disease. The treatment involved delivering therapeutic genes via an adeno-associated viral vector, resulting in improved brain shrinkage and behavioral changes.
A team of researchers found that some cells exhibit random monoallelic gene expression during development, which can affect protein production and potentially lead to diseases. The phenomenon is more common in mature cell types than stem cell precursors.
The European Commission has invested nearly $475 million in 100 gene transfer projects to support basic and clinical research in the field of gene and cell therapy. This funding is aimed at developing new treatments for chronic and rare diseases, as well as novel regenerative medicine approaches.
A GW researcher has discovered that gene therapy can elicit a regenerative response in pig hearts after a heart attack. The treatment utilizes the Cyclin A2 protein to stimulate cellular division and promote cardiac repair.
Joseph C. Glorioso, III, PhD received a Pioneer Award from Human Gene Therapy for his leadership and accomplishments in developing herpes viruses as efficient vectors for delivering therapeutic genes into cells. His research has shown promise for treating complex genetic and acquired diseases, including brain degeneration and cancer.
A large-scale randomized control trial shows Schema Therapy to be significantly more effective than Clarification-Oriented Psychotherapy and treatment as usual in treating a range of personality disorders. The therapy resulted in higher recovery rates, greater declines in depression, and lower dropout rates.
Dr. Ronald G. Crystal developed the first in vivo adenoviral gene delivery vector, accelerating gene therapy translation from lab to clinic. The Pioneer Award recognizes his seminal work on adenoviral vectors.
Researchers have identified a single gene, Ultrabithorax (Ubx), that determines the distinct leg features of worker honey bees, enabling them to collect and transport pollen. The gene plays a critical role in the evolution of social behavior among honey bees, separating queens from workers.
Researchers have developed a gene therapy that improves muscle strength, corrects muscle structure, and prolongs life in animal models of X-linked myotubular myopathy. This devastating disease affects approximately 1 in 50,000 male births, causing severe respiratory difficulties and requiring intensive support.
A recent study published in Human Gene Therapy evaluated the immune response to viral vector delivery systems and their impact on gene therapy's safety and efficacy. The researchers found that the systemic and local immune reactions induced by AAV-based gene therapy did not affect the safety of gene therapy or expression of the replace...
Researchers developed a gene delivery method targeting tumor blood vessels in mice, avoiding healthy tissues. The approach uses a deactivated virus carrying a gene of interest to hijack abnormal blood vessels and alter the tumor microenvironment.
Dr. Katherine High's groundbreaking research in gene therapy has led to novel approaches to correcting hemophilia, with ongoing studies in a current NIH-funded clinical trial. The E. Donnall Thomas Prize recognizes her remarkable contributions to the field, transforming the notion of using genetically engineered mechanisms for treatment.
A new gene therapy has led to a dramatic decline in bleeding events in dogs with naturally occurring hemophilia A. The treatment involves packaging specialized blood platelets with genes that express clotting factor, resulting in significantly fewer serious bleeding events over three years.
A study found that cognitive behavioral therapy for PTSD reduces symptoms and affects underlying biology, reversing changes in gene expression and brain volume. The therapy was associated with improved symptoms and increased hippocampal volume and FKBP5 gene expression.
A single dose of AAV1/SERCA2a gene therapy has long-lasting benefits for advanced heart failure patients, improving heart function status and reducing recurrent cardiovascular events. The study results support the potential use of this gene therapy as a new tool for treating advanced heart failure.
A new gene therapy has shown promising results in reversing heart failure by delivering the SUMO-1 gene directly to the heart. In preclinical testing, the therapy improved cardiac function, reduced heart volumes, and enhanced blood flow compared to other treatments.
Researchers at KU Leuven developed a new protein fusion that combines HIV and MLV replication proteins to create a safer viral vector for gene therapy. This fusion protein reduces the risk of cancer-causing mutations when integrated into human cells, paving the way for more effective treatments for blood diseases.
Researchers at Nationwide Children's Hospital have found a way to overcome the immune system's attack on gene therapy viruses. By using plasmapheresis, a process that temporarily removes antibodies from the body, they were able to increase gene expression in animals with Duchenne muscular dystrophy by 500 percent.
Researchers at the University of Pennsylvania have made an encouraging breakthrough in developing a gene therapy to treat macular dystrophy. They successfully delivered a healthy copy of the BEST1 gene to dogs using viral vectors, showing promise for preventing or restoring vision in individuals with these conditions.
A Mayo-led study has found that sulfasalazine, a commonly prescribed medication for inflammatory bowel disease, fails to reduce diarrhea in patients receiving radiation therapy. The study suggests that the medication may actually increase the risk of diarrhea when used during pelvic radiation therapy.
A study of 70 pediatric patients treated with proton therapy found high local control rates (83%) and overall survival (95%), with subtotal resection correlated to decreased cognitive and endocrine outcomes. Few patients developed growth hormone deficiency, hypothyroidism or hearing loss.
Researchers have found that specific DNA sequences in enhancers affect gene activity, enabling the design of synthetic DNA for gene therapy. This discovery may advance efforts to treat diseases using gene and cell therapies.
Researchers have successfully combined two promising gene therapies to repair muscle damage caused by Duchenne muscular dystrophy and prevent future injury. The treatment, which uses a combination of micro-dystrophin and follistatin, showed significant increases in muscle strength and reduced muscle damage in aged mice.
Researchers at UCLA's Jonsson Comprehensive Cancer Center have successfully combined cellular therapy and gene therapy in a mouse-model system to develop a viable treatment strategy for breast cancer that has spread to the brain. The approach shows significant promise in reducing metastatic brain tumor size, providing new hope for pati...
Researchers have shown that inhalable gene therapy can completely reverse pulmonary arterial hypertension in rat models and reduce expression of SERCA2a, an enzyme critical for calcium pumping. This approach may offer a promising therapeutic intervention for PAH patients.
Researchers have developed a bioengineered decoy that fools the immune system and prevents it from neutralizing the benefits delivered by a corrective gene. The approach could potentially increase the number of patients who can be treated with gene therapy, offering new hope for genetic diseases like hemophilia.
A new gene therapy approach using a partially inactivated lentivirus has shown significant improvement in three children with Wiskott-Aldrich Syndrome. The therapy corrects the genetic defect in blood cells by introducing a normal WASP gene, reducing the risk of cancer-promoting genes.
Researchers have developed a targeted viral therapy that selectively kills breast cancer stem cells and induces apoptosis without affecting normal stem cells. The therapy, which uses the mda-7/IL-24 gene, was shown to be effective in both cell culture and animal models, with no toxic effects on healthy cells.
A gene therapy virus has been approved for treating a rare inherited metabolic disease, and researchers found it rarely integrates into the genome, making it safe. The virus was analyzed in patients and mice, revealing its ability to target mitochondria and potentially correcting genetic defects in human mitochondrial DNA.
Glybera, first gene therapy drug approved in the Western world, was developed after a decade-long search for its genetic mutation. The European Medicines Agency granted marketing approval on November 2, 2012, after several rejections and appeals. Glybera's efficacy was questionable, but its safety was not an issue.
Researchers have developed a new gene delivery method using magnetically targeted nanoparticles that can effectively deliver genes to injured arteries without causing side effects. The technique, which uses stents as a platform for magnetic guidance, shows promise in overcoming current limitations of gene therapy vectors.
Researchers develop a gene therapy that gives mice complete protection against lethal strains of H5N1 and H1N1 flu virus, including the infamous 1918 flu. The treatment involves expressing a broadly neutralizing antibody in nasal epithelial cells, providing substantial reductions in flu replication and virus load.
Recent progress toward developing effective gene therapies for regenerative surgery has shown promising results in growing skin, bone, and other tissues. However, translating experimental gene therapy methods into clinical trials remains a difficult challenge due to technical and cost-effective issues.
Researchers at the University of Missouri have found that the ATP7A gene is essential for copper absorption, leading to a better understanding of Menkes disease. The study used laboratory mice and discovered that the gene's absence can lead to copper deficiency symptoms similar to those seen in children with the disease.
Using two therapies together has been shown to effectively restore vision in dogs with inherited blindness. The treatment approach may have implications for human patients as well. Researchers believe that combining gene therapy and selective photoreceptor cell destruction could lead to better therapeutic outcomes.
A special issue of Translational Research highlights the progress and challenges of gene therapy research, focusing on clinically meaningful studies that combine patient observations with smart experiments. The approach of gene therapy may be applicable to all genetic disorders, offering a promising path for treatment and cure.