Articles tagged with Viral Vectors
A clinical trial of a gene therapy cocktail has shown promising results in treating Canavan disease, a rare and fatal neurodegenerative disorder. The treatment uses an adeno-associated virus to deliver a replacement ASPA gene to the brain, reducing NAA levels and improving symptoms.
Scientists warn of a growing threat from emerging zoonotic diseases as human activities such as land use change, globalization, and social upheaval drive their emergence. The disease burden can be eased by combining efforts between clinicians and public health officials to address the ecological drivers of transmission.
Dr. Samuel Young receives grant to develop critical technology for researching potential therapies targeting LRRK2, a key Parkinson's-related gene. The technology aims to overcome challenges in expressing the gene using commonly used neuroscience research tools.
Scientists at Virginia Tech have discovered a novel antiviral immune pathway in culicine mosquitoes, which produces virus-derived small RNAs similar to piwi-interacting RNAs. This finding suggests a robust immune system, but understanding how viruses overcome it remains an open challenge.
A recent clinical trial in patients with hemophilia B showed that Factor IX gene therapy was able to convert severe hemophilia to moderate or mild disease. The treatment demonstrated a sustained therapeutic effect and eliminated or substantially reduced the need for standard protein replacement.
Researchers at Ohio State University Medical Center have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited form of blindness. The trial aims to treat choroideremia, a disease affecting 100,000 worldwide, and holds promise for other genetic causes of blindness like retinitis pigmentosa.
Researchers used gene transfer to deliver a therapeutic gene that produces protein apoA-1, which removes harmful cholesterol from atherosclerotic lesions. This approach protected against atherosclerosis in rabbit studies, with minimal and stable disease measurements over 48 weeks after a single dose of gene therapy.
Researchers at the University of Pennsylvania have developed a new gene vector, AAV8, that delivers genes to photoreceptor cells more efficiently and safely than a previously used vector, AAV2. The study demonstrates potential for treating eye diseases such as retinitis pigmentosa.
Advances in gene therapy success depend on the development of viral delivery vectors. Researchers have made progress in refining AAV-based vectors, improving their production and use. This has enabled clinical proof of concept and paved the way for commercialization.
Researchers at University of Michigan have developed a novel gene therapy, NP2, that shows promise in providing substantial pain relief for patients with unrelenting cancer-induced pain. The study demonstrates the safety and efficacy of NP2 in reducing pain by up to 80% over four weeks.
Researchers have made significant progress in developing a vaccine against viral hepatitis C, with encouraging immunogenicity and safety profiles shown in phase I trials. The vaccine has induced strong T-cell responses in both treatment-naive patients and healthy volunteers.
A double-blind trial demonstrated a 23% improvement in motor symptoms among patients receiving gene therapy for advanced Parkinson's disease, compared to a 12% improvement in those receiving sham surgery. The study suggests a potential breakthrough in treating this progressive movement disorder.
Researchers have developed an experimental vaccine against the human norovirus, generating a strong immune response in mice. The vaccine uses a novel viral vector-based method and showed promise in inducing high levels of antibodies and a robust white blood cell response in animals.
A new, simplified method for producing large amounts of viral vector cassettes has been developed, overcoming previous limitations. This approach enables efficient packaging of single- or double-stranded AAV vectors and large AAV cassettes, paving the way for commercial production.
A new, fully characterized viral vector is introduced as a reference material to standardize gene therapy protocols in research applications and human clinical trials. The availability of this reference standard allows vector parameters to be expressed in common units, enabling comparison and normalization across laboratories.
Researchers have discovered that RD114 envelope proteins can effectively pseudotype lentiviral vectors, offering an attractive alternative to VSV-G. This technology has the potential to enhance gene therapy approaches by increasing transduction efficiency and stability.
A recent study by The Wistar Institute found that the newly identified human adenovirus AdHu26 commonly infects people, particularly those in Sub-Saharan Africa, rendering it an unlikely candidate as a vaccine carrier. In contrast, chimpanzee adenoviruses demonstrate similar functionality and are considered superior for mass vaccination.
Researchers create nanoparticle that effectively delivers genetic material into cells, outperforming existing methods. The device, a vector, is between 2 ½ and 10 times more effective than other experimental materials, according to the research.
Researchers found that increasing BMPR2 levels reduced pulmonary hypertension and strain on the heart in rat models of the disease. The study suggests that boosting BMPR2 levels could offer a new therapeutic target for treating PAH.
Anna Whitfield, a Kansas State University plant pathologist, will receive a $1 million CAREER Award to study how viruses affect insect vectors. Her research may shed light on viruses that infect vertebrate animals as well as plants.
Veterinary scientists at Penn have successfully used a viral vector to target rod cells in the retina, a critical step towards developing gene therapies for inherited blindness. The study demonstrates the potential of this approach to correct diseases such as X-linked and autosomal dominant retinitis pigmentosa.
A recent study by OHSU researchers explains how cytomegalovirus (CMV) can overcome the immune system's defenses to re-infect individuals. The virus is able to evade a key portion of the immune system called CD8+ T cells, allowing it to re-infect despite a strong and long-lasting immune response.
Researchers have successfully used a gene therapy technique to slow the onset of X-linked adrenoleukodystrophy, a severe brain disease, in two patients. The treatment involved introducing a therapeutic gene into blood cells using a lentiviral vector, which showed promise in slowing disease progression and improving neurological function.
A new study by researchers at the University of California, Davis, and Emory University found that human movement is a crucial factor in understanding disease transmission for mosquito-borne diseases like dengue. The study used satellite-based GPS and culturally-sensitive interviews to track individual human movement, revealing how fin...
A novel adenoviral vector specifically targets the EphA2 receptor on pancreatic cancer cells, improving transduction by 10-fold in vitro. However, in vivo studies show limited targeting to human pancreatic cancer nodules upon injection into mice.
The May issue of Cold Spring Harbor Protocols features a set of methods to analyze protein complexes, including Blue-Native PAGE and differential in-gel electrophoresis. Additionally, researchers can generate viral vectors using adenovirus technology for efficient gene delivery.
Researchers at VIB develop efficient and safe gene delivery approach based on non-viral genetic elements called transposons, overcoming limitations of viral vectors. This technology may simplify gene therapy, improve safety and reduce costs, treating cancer and genetic disorders.
Researchers at Boston University have created a more efficient way to generate induced Pluripotent Stem (iPS) cells using a single lentiviral vector. This breakthrough reduces the risk of genomic integrations and cancer-causing genes, paving the way for human clinical trials.
A study led by Beth Israel Deaconess Medical Center demonstrates a T-cell-based HIV-1 vaccine strategy may be effective in fighting AIDS. The vaccine regimen induced potent immune responses, reducing viral replication and preventing AIDS development in monkeys.
Scientists at UGA create novel synthetic gene vectors that package DNA into nanostructures for efficient gene delivery without triggering immune responses. The study has implications for cancer treatment and vaccine development.
The Indiana University School of Medicine will be home to the nation's sole National Gene Vector Biorepository and Coordinating Center for gene therapy research. The center will help researchers share information and substances to promote discoveries and patient safety.
Researchers at the University of Florida have developed a new version of the adeno-associated virus used in gene therapy that works more efficiently than current vectors. By replacing an amino acid on the surface of the virus, they were able to improve its ability to deliver genes into cells and reduce the risk of triggering an immune ...
Cardiology researchers at The Children's Hospital of Philadelphia have developed a synthetic material that can bind to various gene therapy vectors, allowing controlled local release of therapeutic genes. In an animal study, the new formulation showed significant reduction in restenosis, a hazardous narrowing of blood vessels.
Researchers have discovered a new vector, AAV5, that can carry large genes linked to inherited blindness, paving the way for potential gene therapy treatments. This breakthrough enables the delivery of corrected faulty genes into cells where they are required.
New data shows that vectors derived from AAV5 can accommodate large genes, including those missing in a mouse model of recessive Stargardt disease. This breakthrough has implications for treating individuals with the disease, which is characterized by progressive loss of sight.
A new HIV/AIDS vaccine is being tested in a phase 1 clinical trial at Beth Israel Deaconess Medical Center in Boston. The vaccine uses a replication-incompetent adenovirus vector encoding an HIV-1 envelope gene, aiming to overcome the problem of preexisting immunity to common vaccine vectors.
Researchers found a key protein switch in Chikungunya Virus that allows it to infect and transmit through the Asian tiger mosquito, increasing its ability to spread to new locations. This mutation enables the virus to adapt to areas without typical vectors, posing a global health threat.
The study found that antibodies against arthropod saliva can indicate exposure to bites, allowing for improved assessment of disease transmission risk in endemic areas. This research aims to develop simple and effective prevention tools using immunogenic salivary proteins.
Researchers at the University of Pennsylvania School Medicine have successfully tested a new gene therapy vector that inhibits HIV replication. The treatment, called VRX496, has shown promising results in reducing viral loads and improving immune function in patients with chronic HIV infection.
A Phase I study of novel gene therapy VRX496 for HIV treatment found five patients experienced decreases in viral load and stable or increased CD4 T cell counts after receiving the treatment. The trial also demonstrated the treatment's potential to restore normal immune function against other infections.
Researchers have developed a new vaccine approach using lentivirus that induces a more potent and long-lasting immune response compared to other viral vectors. The approach targets skin dendritic cells, which play a crucial role in recognizing foreign bodies and triggering an immune response.
A vaccine developed by University of Pittsburgh Medical Center researchers has shown 100% protection against the avian flu virus in an animal study. The vaccine, which contains a live virus, stimulates several lines of immunity and is expected to have greater therapeutic value than traditional vaccines.
University at Buffalo scientists developed nanoparticles that delivered genes to adult brain stem/progenitor cells in vivo with no observable toxic effect. The technique may allow repairing brain cells damaged by disease, trauma, or stroke. This breakthrough demonstrates the potential for non-viral vectors in gene therapy.
The UGA/Penn team aims to develop a single-access point for genomic and related information about Apicomplexa parasites, enabling accelerated vaccine, diagnostic, and therapeutic development. The database will link existing databases for Plasmodium species, Toxoplasma gondii, and Cryptosporidum parvum.
Researchers at Pitt University successfully reversed peripheral neuropathy in diabetic mice using gene therapy based on HSV vectors, delivering neurotrophic factors to affected neurons. The approach restores lost nerve endings and prevents progression of neuropathy, with promising results for potential human treatment.
Researchers found that different retroviral vectors from ASLV, HIV, and MLV integrate into human chromosomes at varying sites. The study suggests ASLV may be a promising candidate for human gene therapy due to its distinct integration preferences.
The study found that LR11 plays a crucial role in regulating beta amyloid levels, suggesting its potential as a therapeutic target for Alzheimer's disease. The Emory researchers used lentiviruses to selectively reduce and enhance LR11 expression in mouse brains and cultured cells.
Researchers develop a system to predict West Nile virus risk based on climate factors, including temperature. The model will provide early warnings for public health professionals and serve as a model for other vector-borne disease risks.
Researchers developed a regulatable gene therapy that can be turned on and off using doxycycline, offering potential for treating Parkinson's disease. The treatment uses the glial cell line-derived neurotrophic factor (GDNF) protein to promote survival and differentiation of dopamine neurons.
Researchers at OHSU are developing a new class of viral vaccine vectors, based on cytomegalovirus (CMV), to serve as the basis of an HIV vaccine. The approach aims to generate a superior and more durable anti-HIV immune response that can persist over subsequent years.
Scientists have engineered a vaccine vector that targets mucosal surfaces, which are entry points for many pathogens. The hybrid vector combines features of adenovirus and reovirus to repel viruses like HIV-1 and infectious bioweapons.
Researchers at St. Jude Children's Research Hospital developed a genetic trick adapted from viruses to create gene therapy vectors that can efficiently produce multiple proteins in specific amounts. This technique uses self-cleaving peptides to break down long protein complexes into smaller, functional proteins.
Researchers found that viruses used for gene delivery can influence gene expression, with AAV affecting genes minimally and adenovirus triggering broader responses. The study provides a systematic explanation for the relative safety profiles of two commonly used gene therapy vector classes.
Researchers have discovered a vector using E. coli and Listeria to promote health, with six mice remaining tumor-free for over 90 days after vaccination. The killed bacterial formulation primes the immune system against diseases like cancer.
Researchers successfully treated rats with chemical-induced Parkinson's disease using gene therapy, restoring normal limb movement. The treatment involves delivering two corrective genes into a specific brain region to prime the production of dopamine, which coordinates limb movements.
A NASA-funded study utilizes satellite data on temperature and vegetation to forecast West Nile Virus transmission and identify high-risk areas. The analysis reveals that mosquito populations thrive in warmer, wetter conditions, allowing researchers to pinpoint disease hotspots and make predictions about future outbreaks.
The National Center for Advancing Translational Sciences (NCATS) has established a network to provide clinical researchers with access to human gene vectors and toxicology studies. Seven NIH components will participate in the network, which aims to eliminate barriers to gene transfer research.
A study found that tick-borne diseases are a major concern for travelers, with over 100 viruses and bacteria transmitted through ticks. The most serious problem is posed by hard ticks, which can cause mild irritation but also transmit fatal diseases like Lyme disease and tick-borne typhus.
A genetically stripped down HIV-based vector has been developed that can deliver genes to target cells in the body safely. The vector can also be turned off with a common antibiotic, which suggests doctors may one day be able to control gene expression in people treated with HIV-based gene therapy vectors.
Researchers at University of Iowa have found that a specific gene vector, AAV5, can effectively reach many brain sites following a single injection. The study suggests that AAV5-based vectors could be used to deliver correct copies of genes to cells throughout the central nervous system without multiple injections.