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.
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.
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 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.
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.
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.
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 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.
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.
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.
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 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.
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.
A study published in New England Journal of Medicine shows that physical therapy is just as effective as surgery for patients with meniscal tears and arthritis of the knee. The study found no significant differences in functional improvement between patients who underwent surgery with postoperative physical therapy and those who receiv...
Researchers develop a gene therapy technique to increase enkephalin, an opioid compound, in the bladder wall, reducing pain signals. The treatment approach shows promise for severe chronic pain conditions like bladder pain syndrome/interstitial cystitis.
Researchers develop lipid nanoparticles as vectors in gene therapy to deliver therapeutic genes to cells without degrading within the organism. The technology improves drug absorption for insoluble or poorly soluble molecules, offering a promising alternative for diseases with no effective treatment.
Researchers found local therapy combined with continued EGFR TKI therapy to be well-tolerated and effective in prolonging progression-free and overall survival. Local therapy was associated with a median overall survival of 41 months, a median time to progression of 10 months.
Aravind Asokan, assistant professor of genetics at UNC School of Medicine, received the award for his significant contributions to gene therapy. His lab developed a synthetic viral toolkit to unravel viral infectious pathways and produce novel vectors for gene therapy.
Researchers at Case Western Reserve University discovered a missing RNA element that significantly improves the effectiveness of gene therapy. The element, called Genomic RNA Packaging Enhancer element (GRPE), coordinates the production and filling of genetic material in viral vectors.
Researchers at the University of Texas M.D. Anderson Cancer Center have developed a synthetic gene circuit that can dial up or down gene expression in human cells, enabling refined research for drug resistance and cancer treatment. The circuit's precision will allow scientists to test the boundaries of genes known to confer resistance ...
Researchers from UAB have shown that gene therapy can cure type 1 diabetes in large animals with a single session. The therapy uses a 'glucose sensor' to regulate blood sugar levels, reducing diabetic hyperglycemia and preventing complications.
Researchers in Calgary have launched the world's first gene therapy clinical trial for Fabry disease, a rare inherited enzyme deficiency. The trial aims to transplant stem cells with a working copy of the GLA gene into patients, potentially curing the condition.
A new study from the University of Pennsylvania School of Medicine found that gene therapy improves vision in patients with Leber congenital amaurosis, but also advances retinal cell degeneration. The research provides a more nuanced understanding of the disease and its treatment.
Researchers have successfully treated Duchenne muscular dystrophy in dogs using gene therapy, reducing inflammation and improving muscle strength. The study, published in Molecular Therapy, provides a quantum leap forward in fighting this devastating disease, paving the way for future human clinical trials.
A study published in Cell Reports found that the position of a gene within chromatin affects its expression, contradicting the concept of a singular 'histone code'. The researchers inserted the same gene into 90 different locations in yeast chromosome and discovered significant differences in gene activity.