Researchers have developed a new CRISPR-Cas9 variant that reduces unintended changes in DNA, increasing precision in gene therapy. The SaCas9-HF variant shows high on-target efficiency and nearly undetectable off-target activity, offering a promising alternative for precise genome editing.
Researchers have developed a new approach to delivering sight-saving gene therapy to the retina, using a small needle to inject harmless viruses into the suprachoroidal space. This method could provide a more permanent therapeutic option for patients with common diseases such as wet age-related macular degeneration and inherited retina...
Researchers at RMIT University have developed a non-viral gene delivery method that has proven effective in laboratory tests, opening the door to treatment of various genetic diseases. The method uses Metal Organic Frameworks (MOFs) and could significantly reduce the time and expense involved in bringing new treatments to market.
Researchers at Neuroscience 2019 announce new gene therapy research promising to prevent, treat, and reverse incurable neurological disorders. Successes in restoring lost functions in animal models of neurological diseases are discussed, as well as technique advancements that may improve future treatments.
Boston University School of Medicine researchers have developed a new protocol to produce large quantities of AAVs, which can deliver specific genes into humans and animals. The advanced technique bypasses developmental effects, saving time and reducing the number of animals used in research.
After a tragic death in 1999, gene therapy faced a setback, but James Wilson's rediscovery of adeno-associated viruses (AAVs) paved the way for its resurgence. Today, AAVs are used in nearly 100 drug development programs and have been approved by the FDA for treating a fatal neurological disease.
Researchers have developed a new gene therapy that converts glial cells into neurons, improving motor function in mice and potentially treating stroke. The treatment uses the NeuroD1 gene and has been shown to increase neuronal density and reduce brain tissue loss in mouse models of stroke.
Researchers used a canine model of Leber congenital amaurosis to study the effects of gene therapy on photoreceptor cells. They found that dogs treated before 63% of their cells were lost had lasting improvements, while those treated later experienced continued degeneration.
Researchers at UW-Madison have developed tiny customizable nanocapsules to deliver gene-editing payloads, reducing the risk of unintended genetic edits. The nanocapsules are expected to improve the efficiency and safety of gene therapies, potentially treating inherited diseases and stubborn viral infections.
A new gene therapy has been developed to specifically reduce fat tissue and reverse obesity-related metabolic disease in obese mice by targeting a fatty acid metabolism gene. The therapy resulted in a 20% reduction of body weight and improved insulin resistance and inflammation after just six weeks of treatment.
Researchers at Ohio State University have made significant breakthroughs in gene replacement therapy for Niemann-Pick type A disease, demonstrating its safety and therapeutic effects in nonhuman primates and mouse models. The treatment has shown a 'bystander effect', preventing motor and memory impairment and increasing survival rates.
Researchers used an optimized AAV9 vector to deliver the galactosylceramidase gene to a mouse model of Krabbe disease, improving clinical symptoms and prolonging median survival. The treated mice lived up to 150 days, compared to 41 days for untreated mice.
Children's Hospital of Philadelphia researchers have developed a new AAV vector screening method that captures the full range of gene expression patterns caused by AAV vectors. This innovative technique is expected to significantly advance the field of gene therapy by providing a more sensitive approach to detecting gene transfer sites.
A study by University of Notre Dame researcher Siyuan Zhang and collaborators identified a gene that makes triple-negative breast cancer (TNBC) cells vulnerable to existing therapies. The discovery suggests that a targeted drug therapy may be effective for this aggressive type of breast cancer.
Researchers at the University of Maryland and National Cancer Institute identified 12 distinct gene-pair relationships in tumor cells that could be targeted for cancer therapy. These relationships were associated with varying levels of expression in the two genes and correlated with cancer patient survival.
Scientists at Boston Children's Hospital create human tissue models of CPVT and reverse the condition using gene therapy in a mouse model. The study opens up possibilities for single-dose gene therapy treatments for inherited arrhythmias like atrial fibrillation.
A worldwide coalition of researchers has agreed that light therapy is an effective intervention for preventing oral mucositis in head and neck cancer patients. The new guidelines recommend photobiomodulation therapy, a low-dose light therapy, to prevent the painful ulcers resulting from radiation therapy.
Researchers have developed a genetically modified virus that kills cancer cells and makes them sensitive to chemotherapy drugs, halting tumor progression in mice. The combination of p53 gene therapy and cabazitaxel resulted in full control of tumor growth, with an additive or synergistic effect.
Researchers have successfully developed and tested a gene therapy approach using CRISPR-Cas9 technology to treat Steinert's myotonic dystrophy, a devastating neuromuscular disease. The study showed that the expanded CTG triplet repeat in the DMPK gene was 'cut' and removed from the gene, reducing toxic RNA aggregates in muscle cells.
A team of researchers delivered a therapeutic gene to the spinal canal of infant rhesus monkeys, resulting in sustained expression of the alpha-I-iduronidase enzyme. The study's findings suggest a promising approach for treating severe forms of neuropathic storage diseases.
Shoukhrat Mitalipov, a germline gene therapy pioneer, argues that existing regulations can be strengthened to allow safe treatment. His 17-year-old son Paul also co-authored the editorial, highlighting the importance of understanding gene editing for non-scientists.
A proof-of-principle study shows that gold nanoparticles loaded with CRISPR safely and effectively edited blood stem cells in lab models of HIV and inherited blood disorders. The researchers found that the Cas12a protein partner delivered precise genetic edits, which were maintained for eight weeks after injection.
A study led by Washington University School of Medicine found that proton therapy results in fewer side effects than traditional X-ray radiation therapy for many cancer patients, with similar cure rates. Proton therapy reduced the risk of severe side effects by two-thirds within 90 days of treatment.
A study by Brown University researchers found that stroke patients who receive more physical therapy have lower readmission rates, with those receiving 75 minutes or more of PT being 14% less likely to be readmitted. However, the amount of occupational therapy had minimal impact on readmission risks.
A new study compares the effectiveness and cost of gene therapy to allogeneic hematopoietic stem cell transplantation (HSCT) in patients with major beta-thalassemia. Gene therapy was found to be associated with fewer complications and hospital admissions over 2 years, but was nearly twice as costly.
A small clinical trial shows that gene therapy can safely correct the immune systems of infants with X-SCID, a rare life-threatening disorder. The treatment involves inserting a normal copy of the IL2RG gene into the patient's blood-forming stem cells.
A consortium of researchers identified a rare but important gene target, NRG1, responsible for development of various cancer types. The study suggests that targeting this gene fusion could effectively shut down cancers, offering a new therapy possibility.
Researchers found that older brains with low TRIM9 levels are prone to extensive swelling following stroke, but increasing the gene's expression improves recovery. The study aims to identify what triggers TRIM9 gene expression to develop potential treatments.
A clinical trial found that animal-assisted therapy can foster social competence and increase emotional involvement in patients with brain injuries. The study showed that patients exhibited more active social engagement, expressed positive emotions, and reported higher satisfaction when an animal was present during therapy sessions.
Researchers at Purdue University discovered a molecule called heparan sulfate that can prevent viruses from escaping cells, raising questions about the safety of gene therapy delivery. The study highlights the need to consider how engineered viruses will exit cells to avoid unintended consequences.
A recent study published in The BMJ found that long-term use of oral hormone therapy is associated with a small increased risk of Alzheimer's disease in postmenopausal women. Women who used hormone therapy for over 10 years had an increased risk, especially those diagnosed at age 60 or older.
Dr. Carl June is being honored by ACGT for his lifesaving CAR T-cell treatment, which was the first-ever FDA-approved gene therapy for cancer. The funding from ACGT sustained Dr. June's research, allowing it to continue and ultimately lead to promising treatments.
A new gene therapy using CRISPR/Cas9 targets the accumulation of toxic proteins in progeria syndrome, a rare genetic disorder. The therapy improves health and life span in mice, providing insight into molecular pathways involved in accelerated aging.
Scientists have successfully restored auditory synapse function and hearing thresholds to a near-normal level in adult mice with DFNB9 deafness. The breakthrough uses an AAV-based gene therapy approach that can be used to produce otoferlin and durably correct the profound deafness phenotype in mice.
Newer versions of gene therapy cassettes deliver better performance, increasing muscle strength and protecting against contraction-induced injuries in animal models. The treatment, micro-dystrophin, has been restructured to enhance its functionality.
Researchers have made a breakthrough in treating nerve damage by combining neurosurgical repair with gene therapy, which stimulates the survival of nerve cells and regeneration of nerve fibers. The discovery is an important step towards developing a new treatment for people with nerve damage.
The Okinawa Institute of Science and Technology has unveiled a new web-based tool called ORTHOSCOPE, which quickly analyzes genomic data to estimate gene trees and identify sets of ancestral genes. This allows researchers to infer gene functions and understand species evolution.
Children's Hospital of Philadelphia celebrates European Commission approval of LUXTURNA, a one-time gene therapy treating vision loss due to inherited retinal dystrophy. The therapy has received regulatory approval in both the US and EU, offering new hope for patients with progressive blindness.
Researchers have developed a gene therapy treatment that is reversing disease symptoms in two adults with Sickle Cell Anemia. The treatment, which uses reduced-intensity preconditioning, shows promise for transportability to resource-challenged parts of the world where SCA is most common.
Scientists have discovered that an emerging Parkinson's gene therapy called AAV2-GAD creates new brain circuits associated with improved motor movement. The therapy has shown therapeutic effects for patients by forming new neural pathways in the brain, connecting the subthalamic nucleus to other motor regions.
A study published in Human Gene Therapy found that urocortin 3 gene therapy significantly improved left ventricular function and ejection fractions in mice with induced heart failure. The therapy resulted in a 1.9-fold increase in left ventricular function and a 32% relative increase in ejection fractions.
Researchers developed a combinatorial gene therapy approach that inhibits inflammation and promotes pro-anabolic cartilage genes, preserving articular cartilage better than each approach alone. This strategy was shown to be effective in both moderate and severe post-traumatic osteoarthritis models.
New research presents promising results from preclinical studies using gene therapy to treat amyotrophic lateral sclerosis, Parkinson's disease, and other neurological disorders. Gene therapy has been successfully used to slow disease progression and improve symptoms in mouse models.
A new study by UMass Medical School researchers successfully delivers RNAi-based gene therapy to silence SOD1 protein mutations linked to ALS without adverse effects. The therapy, delivered via a viral vector, achieved silencing of over 90% in some motor neurons, suggesting a safe and potentially one-time treatment for humans.
Researchers at the University of Pennsylvania School of Medicine have discovered that viral vectors used in gene therapy undergo spontaneous changes during manufacturing, affecting their structure and function. The team has developed new ways to prevent these changes, leading to more efficient and safer delivery of gene therapies.
The RAC's elimination would shift review responsibility to local Institutional Biosafety Committees. The new approach aims to reduce redundancies between FDA and RAC roles in human gene therapy.
A team of researchers has developed a gene therapy cure for the Leber Congenital Amaurosis, a genetic form of childhood blindness. The treatment restores vision to treated children and adults, paving the way for new treatments for genetic conditions.
Researchers at Stanford Medicine have successfully developed a method to induce tolerance to gene therapy in mice, eliminating the autoimmune reaction that often occurs. This breakthrough could lead to effective treatments for single-gene disorders such as Duchenne muscular dystrophy.
Gene therapy using optimized AAV to deliver human factor VIII showed substantial hFVIII expression and no detectable antibody response in cynomolgus macaques. The results suggest that AAVhu37-based gene therapy has the potential to advance to clinical trials for treating hemophilia A.
Scientists have developed a novel gene therapy that effectively reduces rhodopsin production and prevents photoreceptor death in dogs with autosomal dominant retinitis pigmentosa. The treatment, which combines shRNA interference with a replacement gene, shows promise for slowing or preventing vision loss in humans.
Researchers at CNIO develop gene therapy with telomerase, proving effective in mice against diseases caused by excessive telomere shortening. The study finds that the gene therapy does not increase the risk of developing cancer, even in a cancer-prone setting.
A study found significant improvement in cone function and no abnormalities in treated eyes for up to six years. The treatment used an AAV5 vector delivered a CNGA3 gene therapy, offering hope for treating genetic blindness.
A new study shows that a single injection of AAVB1-GAA gene therapy prolonged survival and improved enzyme activity in a mouse model of Pompe disease. The therapy also targeted the respiratory system, improving ventilatory measures.
The Alliance for Cancer Gene Therapy (ACGT) is funding innovative clinical approaches to find gene therapy treatments for solid cancers. Recipients of the 2018 research grants include a team working on a vaccine for melanoma, an injectable genetic agent to program T cell receptors, and CAR T cell therapy for brain cancer.
The publication demonstrates the company's technology induces efficient and precise in vivo gene editing using homologous recombination, a natural DNA correction pathway. This early academic research translated into a scalable process for genetic medicines development.
A new study published in Nature Medicine found that fetal gene therapy can prevent and cure neonatal lethal neurodegenerative diseases in humans in utero. The therapy was tested on mice with Gaucher disease and showed promising results, including improved brain degeneration and increased survival time. Researchers believe this approach...
Researchers from UAB successfully used gene therapy to treat type 2 diabetes and obesity in mice by inducing the production of FGF21, a hormone that maintains correct energy metabolism. The treatment led to weight loss and improved insulin sensitivity, reducing the risk of related comorbidities.
Researchers at Washington University School of Medicine have developed a gene therapy method to target damaged kidney cells, which could lead to improved treatment for chronic kidney disease. The approach uses adeno-associated virus (AAV) to deliver genetic material to targeted cells, showing promise in slowing or reversing cell damage.
A new molecular imaging method allows early assessment of gene therapy success, potentially improving treatment for Parkinson's and Alzheimer's diseases. The PET reporter gene/probe system enables noninvasive monitoring of gene expression in all brain areas.
A new gene therapy has been shown to restore hand function in rats with spinal cord injuries by breaking down scar tissue and allowing nerve cell regeneration. The therapy uses a 'stealth gene' switch that can be turned on and off, providing a safeguard and paving the way for human trials.