Articles tagged with Expression Vectors
Kobe University scientists have engineered bacteria to produce a group of compounds with promising pharmacological activities. The breakthrough uses a rational design strategy to create a platform for industrial production of drug candidates.
A Kobe University team has engineered E. coli bacteria to produce the compound pyridinedicarboxylic acid (PDCA) from glucose at unprecedented levels, surpassing previously reported concentrations. The breakthrough enables the clean and efficient synthesis of a biodegradable PET alternative with superior physical properties.
Researchers at the University of Sydney developed a biological 'artificial intelligence' system called PROTEUS, which can accelerate cycles of evolution and natural selection to create molecules with new functions in weeks. The system has potential applications in finding new medicines and improving gene editing technology like CRISPR.
Scientists developed a new technology to produce Cre-loxP organisms in a single step, reducing the need for crossbreeding and decreasing production time. The method involves introducing a TAx9 sequence to prevent Cre gene expression in E. coli bacteria, allowing for precise control and modification of gene expression.
Researchers developed a novel lentivirus-based gene therapy strategy in CD34+ hematopoietic progenitor cells, which showed therapeutic levels of expression of the anti-sickling beta globin protein. Cyclosporin improved transduction efficiency and preserved cell viability.
Researchers studied how viruses move proteins in fruit flies to infect other animals. They found that viral proteins have built-in GPS signals guiding them to precise locations within the host cells. This knowledge could lead to new strategies for disrupting virus movement and controlling insect-borne diseases.
Scientists from Spirovant Sciences describe a novel adeno-associated virus (AAV) gene therapy called SP-101 that has been optimized for efficient human airway cell transduction. After single dose inhaled delivery, the vector showed consistent expression of a functional and regulated shortened human CFTR minigene.
Researchers have shown that repeated administration of lipid nanoparticle-encapsulated mRNA therapy significantly extended survival and reduced serum leucine levels in a mouse model of maple syrup urine disease. The treatment approach may represent a potential long-term universal treatment for MSUD.
Researchers developed a treatment approach using lipid nanoparticles to deliver mRNA therapy for MSUD, extending survival and reducing serum leucine levels. The study also identified a novel AAV variant with desirable biodistribution properties for targeting peripheral organs.
Researchers developed a method to design weaker transcription factors that work together to activate genes without activating naturally occurring genes. This approach, called cooperative assembly, strengthens the factors as a group but weakens them individually, ensuring targeted gene activation and long-term circuit stability.
Researchers developed AEGIS, the first technique to detect backdoor attacks in robust machine learning models. It improves the trustworthiness of artificial intelligence by analyzing robust models and detecting mixed input distributions for poisoned classes.
Researchers found that enhancing NMDAR function via increased serine racemase expression improved attention and cognitive flexibility in middle-aged rats. Upregulating serine racemase in the medial prefrontal cortex also increased glutamatergic synaptic transmission, including NMDAR activity.
Researchers have successfully used AAV1.NT-3 gene therapy to improve muscle physiology and prevent age-related sarcopenia in mice. The treatment resulted in restored muscle mass, strength, and neural connections, offering a potential new option for managing this debilitating condition.
Researchers will use transcriptomics and chemogenetics to identify molecular targets for pain management. The project aims to advance knowledge on pain mechanisms and develop novel therapeutic strategies.
A novel gene therapy has shown sustained expression of clotting factor VIII, leading to a reduction or complete elimination of bleeding events in patients with hemophilia A. The trial demonstrated improved production of coagulation factor VIII over prolonged periods.
Researchers at Children's Hospital of Philadelphia have developed a system that can fine-tune protein expression from gene therapy vectors, addressing the need for controlled dosing. The 'dimmer switch' uses alternative RNA splicing and an orally available small molecule to adjust levels of expression up or down as needed.
Researchers have developed an immune tolerance platform called ImmTORTM to overcome challenges in gene therapy, including immunogenicity and durability. The addition of ImmTOR nanoparticles to AAV vectors has shown potential to enhance efficacy, safety, and durability by mediating more efficient transgene expression.
Researchers have developed a new approach to prevent toxicity seen in sensory neurons of dorsal root ganglia after gene therapy to treat neurological disorders. The approach involves modifying a transgene with a microRNA target, which reduces transgene expression and eliminates toxicity.
Wake Forest Institute for Regenerative Medicine scientists have developed a faster and more efficient gene editing tool using the CRISPR/Cas9 system. The new delivery system packages both essential components together, enabling transient Cas9 expression and avoiding unwanted results.
Researchers at Massachusetts General Hospital found that age itself facilitates the spread of Alzheimer's-disease-associated protein tau in the brain. The study also identified increased vulnerability in specific brain regions, particularly the entorhinal cortex, which is where Alzheimer's-related tau pathology first appears.
Researchers have created a new CRISPR/Cas9 delivery system that reduces off-target effects and improves gene editing efficiency. The 'hit and run' approach delivers Cas9 activity transiently, allowing for highly efficient genome editing while minimizing unwanted results.
A new carrier to transfer genes into plants has enabled crop scientists to study traits and diseases in wheat and maize more quickly and easily. The Foxtail mosaic virus (FoMV) has overcome limitations of existing carriers, allowing for the expression of a wide range of proteins in host plants.
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 Japanese research group has developed a CRISPR/Cas9 system to target and block HIV-1 production in infected cells. By targeting two regulatory genes tat and rev, they were able to significantly lower the expression and functions of both genes.
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.
Researchers have developed virus-derived expression vectors as a potential gene therapy vehicle for treating various diseases. These vectors use viral sequences to silence specific genes or induce RNA interference, effectively downregulating viral replication and expressing therapeutic proteins.
A new study identifies optimal adeno-associated virus (AAV)-based gene therapy delivery vector constructs to treat Hemophilia A. The researchers successfully improved the design of AAV vectors, demonstrating significant and differing effects on liver-specific expression of the human factor VIII transgene in mice.
Researchers developed a mini-intronic plasmid (MIP) system that can enhance transgene expression by up to 40-100-fold when used with adeno-associated viral (AAV) vectors. This could reduce the cost of gene therapy and lessen immune responses.
Researchers have found that the length of Klf4 mRNA affects iPS cell reprogramming, with shorter forms leading to incomplete reprogramming. Longer forms result in more complete reprogramming and higher protein expression levels.
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.
Gene therapy successfully replaces protein missing in Pompe disease when targeting liver cells, reducing immune system reaction. Combining liver-expressing vector with ubiquitously expressing vector boosts overall effectiveness of the treatment.
Gene therapy using stem cells holds great potential for treating a range of diseases, thanks to advances in gene marking techniques, PCR sequencing, and chromatin insulators. The success of these methods could lead to improved patient survival rates.
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.
Researchers at Yale University have developed a method to target specific populations of nerve cells in the brain using viruses. By injecting an adeno-associated virus into the brain, they can trigger the expression of a jellyfish gene that glows green in certain neurons. This approach has significant potential for the treatment of neu...
A study by Penn researchers found that a common gene delivery system for muscular dystrophy can trigger an immune response in mice unless paired with a muscle-specific promoter. The use of restricted promoters is crucial before expanding clinical trials.
Researchers at Memorial Sloan-Kettering Cancer Center and Vion will study the use of TAPET bacterial vector technology for tumor diagnostic imaging in preclinical animal models. The goal is to define molecular signatures that result in preferential accumulation of TAPET bacteria in tumors, enabling more effective diagnosis.
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.
The NIH granted Vion Pharmaceuticals a two-year, $750,000 Small Business Innovation and Research (SBIR) grant to enhance the anti-tumor efficacy of its TAPET bacterial vector. The goal is to deliver potent anti-cancer agents directly at the tumor site, leveraging TAPET's preferential replication in tumors.
A team of UI researchers has advanced understanding of how gene therapy might effectively treat cystic fibrosis by addressing two fundamental problems: inefficient gene transfer and lack of gene persistence. They demonstrated efficient and lasting gene transfer into airway cells using a new vector from the retrovirus family, feline imm...
A University of Iowa research team found a novel approach to enhance binding between vectors and cells, making gene transfers up to 10 times more efficient. The breakthrough could lead to better methods for treating diseases like cystic fibrosis.
Researchers identified a gene called Irx-4 that helps maintain the boundaries between heart chambers. The discovery opens up new avenues to understand heart chamber formation and may also shed light on the cause of certain heart defects.
University of Michigan scientists have developed a new generation of viral vectors that deliver the dystrophin gene to the muscles of adult mice with muscular dystrophy. The new vectors, called 'gutted' viruses, are stripped of most of their original genes to make room for the large dystrophin gene and reduce immune response.
Researchers at Washington University School of Medicine have created a way to create harmless vectors from harmful viruses. They showed that the vectors are efficient couriers of genes and can be used to study gene regulation and functions, as well as deliver DNA vaccines.
Researchers at the University of Pittsburgh have constructed a novel delivery system for gene therapy of liver disorders, using a reconstituted chylomicron remnant (RCR) that can safely transport genes to target cells. The system has resulted in extended production of therapeutic proteins in animal models.
Researchers at the University of Pennsylvania School of Medicine discovered that nerve growth factor induces cell death in some childhood brain tumor cells. The findings suggest a potential new treatment approach for these cancers, expanding scientists' understanding of NGF's role in development.
Dr. Neal Lane raises questions about dumping of Japanese vector supercomputers in the US market, affecting domestic industries, and calls for swift investigation by federal agencies like Commerce and International Trade Commission.