Articles tagged with Viral Vectors
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Researchers compared six adeno-associated viral (AAV) vectors for efficiency of gene transfer to both retinal pigment epithelial and photoreceptor cells. Human pluripotent stem cell-derived organoids were used as an in vitro test system for AAV vector development.
A new environmental monitoring project has found increased numbers of deer ticks carrying Lyme disease in Southern Indiana. The IU Environmental Resilience Institute and Prepared for Environmental Change Grand Challenge launched a project to analyze ticks and mosquitoes for diseases that are dangerous to people.
Scientists warn of increased infectious diseases transmitted through vectors in the coming decades. The Asian tiger mosquito, introduced via trade and tourism, has not yet filled its ecological niche globally, posing a threat to regional health.
A new gene therapy approach has been shown to reduce the need for blood transfusions in patients with severe beta-thalassemia. The treatment, known as LentiGlobin BB305 vector, was effective in eliminating the need for transfusions in 73% of patients, paving the way for potential treatment.
Researchers found that gene therapy using adeno-associated viral vectors can only temporarily alleviate congenital adrenal hyperplasia (CAH) by replacing the defective gene. A long-term solution requires targeting genetic mutations in adrenocortical stem cells, according to a study published in Human Gene Therapy.
Researchers discovered a potential way to prevent mosquito-borne diseases like Zika and dengue by targeting the sphingolipid pathway in the midgut. This approach could stall viral replication and prevent transmission to humans.
Researchers at the University of Guelph have made a breakthrough in combating Ebola with an innovative antibody delivery method. The approach delivers monoclonal antibodies through a viral vector, bypassing the need for a natural immune response, offering 100-per-cent protection against Ebola infection in mice.
A study in São Paulo found that mosquitoes that transmit diseases are adapting to urban environments, thriving in smaller green spaces where other species disappear. The research highlights the need for further study on how habitat loss affects vector insects and disease transmission.
A special issue of Human Gene Therapy highlights Chinese research advancements in gene and cell therapy, including novel viral and nonviral vectors for gene delivery. The articles explore innovative applications of CRISPR technology in various disease areas.
Researchers found liver and neuronal toxicity with high doses of gene therapy delivered using an adeno-associated virus (AAV9) vector. The study warns about the importance of prioritizing patient welfare in gene therapy research.
A recent study found that tRNA fragments in mosquitoes can modulate their ability to transmit viral diseases to humans. The researchers identified 55 tRNA-derived fragments with varying expression patterns across different strains and developmental stages.
Researchers developed a novel gene delivery approach using scaffold/matrix attachment region (S/MAR) to enhance long-term transgene expression in proliferating cells. The modified vectors were able to establish colonies and maintain stable transgene expression in HeLa cells.
A recent study by the Smithsonian Tropical Research Institute and other institutions reveals that conserving old-growth tropical rainforests is crucial for preventing new outbreaks of mosquito-borne diseases. The research found that disease-carrying mosquito species are scarce in undisturbed forest areas compared to disturbed ones.
Researchers at Caltech have developed two new viral vectors that can deliver genetic cargo to neurons in the central and peripheral nervous systems. The vectors, AAV-PHP.eB and AAV-PHP.S, can reach their targets via the bloodstream and are customizable for gene therapy applications. They offer a significant improvement over existing me...
A new Zika virus vaccine has shown 100% protection in mice, utilizing the NS1 protein. The vaccine's effectiveness was demonstrated through a single-dose immunization strategy.
Cases of human Powassan virus infection are rising in the US, particularly in northeastern and midwestern states. The virus is transmitted by Ixodes ticks and can cause fatal neuroinvasive disease and long-term neurological effects.
A new review discusses how gene therapy delivered via adeno-associated viruses (AAV) can trigger an immune response in T cells, leading to activation and exhaustion. The review highlights the variability in individual patients' immune responses to AAV vectors and potential strategies to suppress these reactions.
Researchers envision AAV as a platform for delivering novel tools for genetic manipulation, including CRISPR-Cas9 and RNA interference. The combination of tailored delivery vectors and new editing techniques will enable unique approaches to therapeutic gene expression.
New research concludes that AAV vectors used in gene therapy do not increase the risk of cancer and may even have anti-tumorigenic properties. The study's authors review biomedical evidence showing that AAV2 can negatively impact other viruses associated with malignancy.
The Cornell University-led Northeast Regional Center for Excellence in Vector Borne Diseases aims to educate a cadre of vector biologists and public health practitioners through new courses and a master's program. The center will conduct applied research to prevent, control, monitor, track and respond to vector-borne disease outbreaks.
Researchers at UC Riverside have identified a novel strategy for controlling viral diseases by targeting the interaction between a viral effector protein and a host hormone. The study found that the cucumber mosaic virus manipulates plants to release odors attractive to aphids, which transmit the virus.
Gene therapy for liver disease is advancing with the help of adeno-associated viral (AAV) vectors, offering new therapeutic possibilities. The article discusses preclinical and clinical studies that have led to a better understanding of immune responses to AAV gene therapy.
Researchers at the University of Pennsylvania have developed a dual gene therapy approach using CRISPR/Cas9 to deliver key components to treat hemophilia B. The study showed stable Factor IX activity in newborn and adult mice, with all treated mice surviving a partial liver removal without complications.
A WSU researcher has developed a modified viral vector that reduces the risk of cancer cells and can be used for multiple blood diseases. The new delivery system is being translated into a stem cell gene therapy to treat a life-threatening immunodeficiency in newborns.
Researchers developed efficient lentiviral and adeno-associated virus-based approaches to deliver CFTR genes to the airways of pig models with cystic fibrosis. These methods resulted in functional CFTR expression, improving early cystic fibrosis pig phenotypes.
Researchers have identified Zika virus in mosquito species other than Aedes aegypti, raising concerns about alternative transmission sources. However, studies show that Culex species mosquitoes are highly unlikely to transmit the infection to humans, highlighting the need for targeted mosquito control efforts.
A new gene therapy approach successfully delivered a therapeutic gene to the brains of treated mice, restored enzyme function, and extended survival. The treatment holds promise for developing similar therapies for humans and targeting additional brain disorders.
The yellow fever virus, transmitted by Aedes aegypti mosquitoes, is a significant public health threat to the Americas. The recent resurgence of YFV in Africa and Asia, combined with vaccine shortages, highlights the need for urgent measures to prevent infection and control its spread.
A novel gene therapy developed at St. Jude Children's Research Hospital has shown promising results in rebuilding the immune system of young adults with X-linked severe combined immunodeficiency disease (SCID-X1). The treatment, which combines lentivirus gene therapy with busulfan conditioning, resulted in improved health and reduced c...
A phase 1 trial evaluated two candidate Ebola vaccines, Ad26.ZEBOV and MVA-BN-Filo, in healthy volunteers. The study found that immunization with Ad26.ZEBOV generated an initial immune response and sustained elevation of specific immunity, suggesting partial protection against Ebola.
A new study in patients with methylmalonic acidemia found lower than expected levels of antibodies against adeno-associated viral vectors, suggesting encouraging implications for gene delivery as a treatment. The absence of AAV antibodies in most patients who lack the deficient enzyme has promising results for gene therapy.
A study has created global maps showing the geographical limits of chikungunya and dengue viruses, highlighting a rapid expansion in chikungunya's global extent. The research emphasizes the need for improved diagnostic tests and clear protocols for controlling both diseases.
ESTEVE has developed two new investigational gene therapies, EGT-201 and EGT-301, to treat Sanfilippo B syndrome and Hunter syndrome. The treatments join a promising gene therapy platform aimed at restoring enzyme function in patients with severe and debilitating rare diseases.
Recent gene therapy developments focus on viral vectors like AAV for safe gene transfer, and new gene editing tools offer targeted gene inactivation or insertion. These approaches aim to treat multiple diseases with platform strategies.
Scientists have successfully delivered a CRISPR/Cas9 therapeutic to adult mice with Tyrosinemia type I, correcting 6% of the mutated FAH gene in liver cells. This breakthrough uses AAV and lipid nanoparticle delivery mechanisms, paving the way for potential human treatment.
A new study reveals a direct relationship between Alzheimer's disease and brain cholesterol levels. Increasing the enzyme CYP46A1 may help eliminate excess cholesterol from the brain, correcting amyloid plaques and degenerated tau protein. This discovery opens up a new line of research for Alzheimer's treatment.
Researchers have recreated the evolutionary lineage of AAVs to reconstruct an ancient viral particle that is highly effective at delivering gene therapies. The approach could lead to designing a new class of genetic drugs that are safer and more potent than those currently available.
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.
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.
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.
Chikungunya's Asian tiger mosquito relies on road networks to disperse, posing a challenge for disease control efforts. The study suggests fumigating vehicles at checkpoints could prevent the spread of the vector and reduce its impact on human populations.
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 have successfully developed an oral vaccine delivery system using Streptococcus mitis to induce mucosal immune responses in the mouth and beyond. This innovative approach has potential to protect against diseases such as tuberculosis, AIDS, and intestinal infections, making it a promising tool for global health threats.
Researchers used landscape genetics to study Ae. albopictus dispersal in the US, finding it traveled by 'jump' dispersal via cars and trucks along highways, with wetlands supporting mosquito growth. The discovery highlights human-aided gene flow's role in accelerating species adaptation.
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.
Researchers have successfully tested a new vaccine regimen that provides rapid protection against Ebola virus in monkeys, with some animals remaining protected for up to 10 months after vaccination. The experimental ChAd3 Ebola vaccine demonstrated strong immune responses in both single-dose and prime-boost regimens.
A novel HIF-1α viral vector effectively reduces apoptosis in primary cultured hippocampal neurons exposed to amyloid-beta protein. This breakthrough may lead to the development of gene therapy as a potential clinical treatment for Alzheimer's disease.
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. 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 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.
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.
Researchers found that a gene therapy approach using rAAV vectors can promote a regulatory immune response, allowing for sustained gene expression in muscle tissue without the need for immunosuppression. The study used M-type α-1 antitrypsin (M-AAT) and demonstrated long-term expression in patients with AAT deficiency.
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 study analyzed West Nile virus epidemics in Dallas County, Texas, finding that they begin early after warm winters and are predicted by the mosquito vector index. The analysis also revealed higher incidence rates of neuroinvasive disease in recent years compared to previous decades.
Researchers analyzed decade of West Nile infections, weather, and housing data to identify optimal time for intervention. The study found that using a mosquito vector index can nip outbreaks in the bud, but late intervention may still be necessary if data are unavailable.
Researchers at OHSU have discovered a strategy to increase the number of viral peptides that T cells recognize, allowing them to more effectively respond to HIV. The method uses cytomegalovirus (CMV) to generate SIV-specific T cells that can target multiple viral peptides, providing a better targeting system for the immune system.
Researchers have created a novel vaccine candidate for Chikungunya virus using large-scale codon re-encoding, exhibiting stable phenotype and reduced viral fitness. The approach could lead to the rapid design of next-generation viral vaccines against emerging viral pathogens.
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 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.