Articles tagged with Gene 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...
Researchers have developed a new reprogramming factor cocktail that produces high-quality induced pluripotent stem cells with fewer genetic abnormalities. The SNEL combination outperforms existing methods, such as OSKM, in terms of cell quality and efficiency.
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.
A new gene therapy approach has been shown to be effective in preventing and treating botulism exposure, with a single treatment leading to prolonged production of antitoxin proteins. The therapy, known as VNA gene therapy, can protect against toxin-mediated diseases such as C. difficile and Shiga toxin-producing E. coli infections.
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.
Researchers at UNC Lineberger Comprehensive Cancer Center have developed a new integrated approach to pinpoint genetic drivers of cancer, identifying eight genes linked to luminal breast cancer. These findings offer hope for personalized medicine and potential targeted therapies.
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.
Research at VHIO confirms Myc inhibition as a sound and effective therapeutic strategy for glioma, with no evidence of resistance or side effects. The study reveals new insights into the biology of Myc and its potential to halt normal cell division, leading to tumor cell death.
Scientists at Mayo Clinic and Scripps Research Institute developed a new therapeutic strategy to combat the most common genetic risk factor for ALS and FTD. They discovered a potential biomarker that can track disease progression and measure the efficacy of therapies.
A team of researchers at the University of Texas at Arlington has developed a genetic computer network model that can predict the onset of mental illnesses such as bipolar disorder and schizophrenia. The model uses single nucleotide polymorphism networks to analyze a patient's genetic pattern and apply personalized therapy.
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.
Scientists have discovered a new form of dystrophin protein that can be produced through an alternate cellular mechanism in patients with Duchenne muscular dystrophy. This mechanism involves an internal ribosome entry site and could offer a novel therapeutic approach for patients with mutations affecting the first four exons.
Researchers successfully corrected disease-causing mutations in cells from patients with beta-thalassemia using CRISPR/Cas9 technology. The corrected cells showed restored expression of hemoglobin and could differentiate into mature blood cells.
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.
A genetic screening test can predict which children with one of the most common childhood kidney diseases will respond to standard therapies, guiding treatment and improving clinical outcomes. The test was found to be more predictive than a kidney biopsy in identifying non-responders to immunosuppressive treatments.
A combination treatment of gene inhibitor AAVshPTEN and salmon fibrin injections restored voluntary motor function impaired by spinal cord injury in rodents. This breakthrough expands on previous research at UCI and provides a novel scaffold for neuronal axons to grow and link up again.
Researchers from Scripps Florida have discovered a gene called Spns1 that plays a critical role in combating aging. The study found that manipulating the function of this gene can affect both developmental senescence and long-term biological aging.
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.
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 investigate gene therapy as a potential treatment to improve viability and regenerative capacity of injured adult retinal ganglion cells. Studies using modified viral vectors introduce genes into injured visual pathway cells, aiming to promote long-distance axon regeneration.
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 at Scripps Research Institute found that the drug rapamycin can improve the delivery of genes to blood stem cells, increasing efficiency from 30-40% to up to 80%. This breakthrough could lead to more effective and affordable treatments for leukemia and sickle cell anemia.
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.
Researchers discover distinct mutational signature and nine significantly mutated genes associated with nasopharyngeal cancer. The findings provide an enhanced road map for studying the molecular basis of this disease, which has limited understanding compared to other cancers.
Researchers have designed RNA aptamers that specifically target and inhibit PAI-1's anti-clot-busting activity. These aptamers demonstrate the potential for blocking PAI-1-associated vascular events, offering a novel therapeutic option for cardiovascular disease prevention.
Researchers at Scripps Florida have discovered how a specific genetic mutation damages the developing brain, leading to intellectual disability. The study suggests new possibilities for therapeutic intervention and highlights potential biomarkers for cognitive failure.
Amino acids at positions 11, 71 and 74 on the HLA-DRB1 gene identify RA patients at risk of joint damage and early death. The findings suggest that genotyping can predict disease outcomes in rheumatoid arthritis patients.
A nationwide study, led by the Children's Hospital of Eastern Ontario Research Institute, has solved 146 rare disorders and identified 67 novel genes associated with rare diseases. The research team used exome sequencing to identify common biological pathways across multiple rare disorders.
The study identified oncogenic driver genes in over 1,000 lung cancer patients and found that those with targeted treatments survived longer. The researchers aim to expand this technology to other types of lung cancer.
A study has identified 152 unique genes that may be responsible for keloid scarring, a condition characterized by raised, firm skin areas. The researchers found that certain genetic pathways play a crucial role in the development of keloids and could lead to new treatment options.
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.
A study found that genetic alterations in lung cancer tumors can help select targeted treatments, resulting in improved survival rates for patients. The researchers identified actionable oncogenic drivers in 64% of patients and used this data to guide treatment decisions.
Researchers have developed gene therapies to prevent and treat blinding diseases, including choroideremia and retinitis pigmentosa. These treatments involve replacing missing genes in the cells at the back of the eye, resulting in improved vision for some patients.
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.
A study has identified a dozen genetic mutations involved in changes to complete blood counts and the onset of severe biological disorders. The research used DNA from 6,796 people and found mutations in genes related to red and white blood cells and platelets.
Researchers used a genetic approach to find that cortical neurons play a key role in initiating the disease, while shutting down mutant huntingtin in both sets of cells corrected symptoms. The study suggests new targets for therapeutic drugs to slow the devastating disease.
Researchers at UNSW Australia have successfully regrown auditory nerves using bionic ear technology and gene therapy, potentially improving outcomes for people with cochlear implants. The technique delivers neurotrophins to cells near the implant electrodes, promoting nerve regeneration.
A majority of Europeans view biomedical research as a source of hope and trust the public to challenge opinion. However, most lack scientific literacy and rely on experts for understanding, highlighting the need for education and communication about research findings.
A meta-analysis of randomized controlled trials found that chemotherapy improved progression-free survival compared to epidermal growth factor receptor tyrosine kinase inhibitors in patients with wild-type EGFR tumors. However, overall survival did not differ between the two groups.
A gene therapy approach using adeno-associated virus (AAV) successfully treated heart disease in mice with Friedreich's ataxia, restoring full functionality to the hearts. The treatment, which introduced a normal copy of the FXN gene, prevented the development of heart disease and even fully cured advanced-stage cases.
Researchers aim to deliver GDNF into muscle cells using a viral vector to protect motor neurons and slow disease progression in a rat model of ALS. The study, led by Cedars-Sinai Regenerative Medicine Institute, could pave the way for a clinical trial if successful.
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.
Researchers at Tel Aviv University have discovered that DNA therapy can preserve inner ear nerve cells in humans with certain types of progressive hearing loss. The study uses a protein growth factor to block degeneration and has important implications for enhancing sound perception with cochlear implants.
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...
Researchers found that patients with specific gene variants exhibited a significantly lower risk of experiencing relapse after treatment for chronic hepatitis C. The study, which involved over 300 patients, suggests that these variants may play a role in preventing the virus from becoming unstable and causing recurrence.
Researchers at Sanford-Burnham and Icahn School of Medicine at Mount Sinai have discovered a key cellular process leading to heart failure, which can be halted with a therapeutic approach. Blocking the effects of miR-25 has improved cardiac function and survival in mice.
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.
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 research team developed an adjuvant that optimizes lentivirus gene transfer by enhancing virus attachment to target cells, resulting in a three-fold increase in transduction rate. This improvement reduces the need for additional viruses, potentially leading to more effective treatments for genetic disorders.
Johns Hopkins researchers have identified a set of genes that can be turned back on to evade immune detection in ovarian, breast, and colorectal cancer. Reversing these epigenetic changes with an FDA-approved drug allows cancer cells to become more vulnerable to immune therapy, potentially leading to more effective treatment.
A study by Michigan State University researchers used rats and vanilla frosting to identify genetic factors contributing to binge eating. The Sprague-Dawley rat strain was found to be prone to binge eating, narrowing the scope of possible genes involved in the disorder.
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.
Researchers have discovered that genes expressed in the nasal passages can serve as accurate proxies for those expressed deeper in the lungs. This breakthrough allows for less invasive and more affordable genetic profiling of asthmatic patients, potentially leading to improved treatment options and better understanding of the disease.
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.
Duke researchers have successfully used gene therapy to induce stem cells to produce growth factor proteins, overcoming the challenge of delivering these proteins after implantation. The technique allows for long-term delivery and could be applied to various orthopedic tissues, presenting a significant step toward commercialization.
A team of researchers at the University of Montreal has discovered a promising new approach to treating leukemia by targeting the Brg1 gene. The study found that removing or inhibiting this gene can permanently shut down cancerous cell growth and division.
Hal Dietz, a Johns Hopkins pediatric cardiologist and geneticist, has been recognized for his groundbreaking work on Marfan syndrome. He identified the cause of the rare genetic disease and found that losartan can attenuate overgrowth of the aorta.
Researchers use antisense oligonucleotides to skip over the mutated exon causing Huntington's disease, preventing formation of toxic protein fragments. The innovative therapeutic strategy is a proof-of-concept for treating complex diseases.