We narrowed to 497 results for: RAP
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TypeCollection... frequently use AAVs to develop CRISPR-based therapeutics. One limitation of AAV vectors is that the packaging...
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AAV Purification by Iodixanol Gradient Ultracentrifugation
TypeProtocol... to collect the next fraction. The flow will be rapid at first and will progressively slow down. Option...methods improves infectious titer and yield." Gene therapy 6.6 (1999): 973-985 (Link opens in a new window... Rist Wolfgang, and Lamla Thorsten. Human Gene Therapy Methods. August 2015, 26(4): 147-157. doi:10.1089... -
Fluorescent Protein Guide: Empty Backbones
TypeCollection... : Entry by Robert Campbell Spectra Database : Graphing site hosted at the University of Arizona Shaner... -
Genetic Code Expansion
TypeCollection...and evolve proteins for various purposes from therapeutics to biopolymers. Tips for Success Before beginning... -
Cancer and the Immune System: Deciphering the Relationship
TypeBlog Post...Existing therapies and effects on immune system Conventional cancer therapies include radiation therapy (owing...Curie), chemotherapy, and surgical removal of the tumor. However, these conventional therapies produce... Types of immunotherapeutics In lieu of the cancer-immune system interactions, therapeutics have been ...beneficiaries. Collaborative therapy For the most efficient outcomes, collaborative therapies must be explored. ...probability of a relapse. Chemical and radiation therapies cause a transient shutdown of the immune system...boost the activity of the immune system, the therapeutic ability of even these conventional antitumor ...proteins are one of the most commonly used immunotherapeutic agents. Currently the FDA has 12 approved ... -
CRISPR in the Clinic
TypeBlog Post...Current CRISPR Therapies Cancer and CAR-T therapy The FDA has already approved CAR-T immunotherapy for cancer...CRISPR therapies CRISPR technology is rapidly evolving, and many of the challenges with therapies involve...delivery and editing strategies for CRISPR therapies. Systemic therapies involve CRISPR-Cas delivery directly... initiated a CRISPR therapy for hATTR with the first-ever systemic CRISPR therapy. They engineered a CRISPR-Cas...this therapy is affecting hATTR patient outcomes. Bacterial infection and HIV Most CRISPR therapies have...sponsored by Excision Biotherapeutics. Additional ongoing trials The CRISPR therapies don’t stop with the...longer an accurate descriptor; progress in clinical therapy is more fitting, so it is time to update our post... -
Mesothelioma - Causes, Symptoms, and Treatment
TypeBlog Post...treatments, such as surgery, radiation, and chemotherapy, immunotherapy is coming to the fore as a cutting edge...form of treatment known as hyperthermic intraperitoneal chemotherapy (HIPEC). With this technique, surgery...performs a heated chemotherapy wash throughout the abdominal cavity. This targeted chemotherapy approach has...susceptible to up and coming immunotherapies. The standard definition of immunotherapy is the use of drugs (or...research opportunities are being provided to immunotherapy than ever before. Although different types of...great promise in up and coming treatments like immunotherapy. Mesothelioma Mesothelioma is a terrible disease...cancerous masses (tumor resection), systemic chemotherapy to kill cancerous cells that may have spread... -
Meet the New Addgene Board Members!
TypeBlog Post...employees at Dimension Therapeutics, she helped build and grow the gene therapy division of the company...journal Molecular Therapy. Vandenberge is also the Grousbeck Family Chair in Gene Therapy, Director of the...Khandan Baradaran’s expertise in viral safety, gene therapy vector development, vaccine development, and assay...Addgene. She was Head of Quality at Dimension Therapeutics, which was acquired by Ultragenyx Pharmaceutical...Stegmeier is the Chief Scientific Officer at KSQ Therapeutics and is an Addgene depositor. KSQ uses a CRISPR-Cas9...crises like those and ended up in the field of gene therapy.” He’s also worked on vaccines throughout that ...a variety of commercialization efforts in gene therapy either through direct industry collaborations, ... -
Targeting HIV-1 with CRISPR: Shock and Kill or Cut it Out?
TypeBlog Post...challenge for CRISPR HIV-1 therapies is sequence specificity. Whereas antiviral therapies target HIV-1 at the...Today, HIV-1 can be controlled with antiviral therapies such that the virus is undetectable in the blood...demonstrated two potential strategies for CRISPR-HIV therapeutics - one using dCas9-SAM to activate HIV-1 transcription...untreated individuals. Symptoms of AIDS include rapid weight loss and an increased risk of infection, ...usually seen in healthy individuals. Antiretroviral therapy (ART) can virtually eliminate plasma HIV-1, improving...the probability of off-target effects. In such a therapy, CRISPR/Cas9 would need to 1. excise the HIV-1 ...most HIV-1 patients. Obstacles for CRISPR HIV-1 therapy Both of these methods represent exciting advances... -
Viral Vectors 101: AAV Serotypes and Tissue Tropism
TypeBlog Post...human gene therapy. Molecular Therapy: The Journal of the American Society of Gene Therapy, 14(3), 316...AAV1 was the first viral vector approved for gene therapy (Issa et al., 2023). It may be a good choice for...high transduction efficiency in multiple tissues, rapid transgene expression, and can cross blood vessel...rAAV treatment, which can pose problems for gene therapy applications. Cross-reactivity of AAV serotypes...AAV7, and AAV8 in the Mouse and Rat. Human Gene Therapy, 19(12), 1359–1368. https://doi.org/10.1089/hum...evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous...underexplored sites on the AAV capsid. Molecular Therapy. Methods & Clinical Development, 26, 331–342. https... -
Science as Art
TypeBlog Post...scientist and photographer based at the University of Queensland, whose outstanding marine photography has attracted...” Daniel went on to discuss his work: “Macro photography has been my passion for a while - a tool to expand...life cycle of the creature they deal with.” So photography can be used as a way to better understand and...scientist and non-scientist alike. “The other thing photography has shown to me - it requires an awful amount...data, publish in time, defend the thesis, etc. Photography is critically important in many fields of biology... can be found on his website http://www.microworldsphotography.com/. Another artist who spent his life...acclaim - including being featured in the National Geographic and Smithsonian Magazine, where the editor stated... -
Important Considerations When Using AAVs
TypeBlog Post...Future of rAAV gene therapy: Platform for RNAi, Gene Editing and Beyond." Human Gene Therapy ja (2017). PubMed...being made to make them even more useful for therapeutic purposes. The University of Pennsylvania and ...These vectors can be used to improve targeted therapies for the eye, skeletal muscles, and the central...Cas9 but have the potential to deliver other therapeutic molecules such as microRNAs (miRNAs), short hairpin...insertional mutagenesis, and cancer." Molecular Therapy 15.10 (2007): 1740-1743. PubMed PMID: 17882145....tropism in mice after systemic injection." Molecular Therapy 16.6 (2008): 1073-1080. PubMed PMID: 18414476. ...anterograde and retrograde axonal transport." Human gene therapy 25.8 (2014): 705-720. PubMed PMID: 24694006. PubMed... -
Predicting Adverse Reactions to Monoclonal Antibody Drugs
TypeBlog Post...Monoclonal antibody drugs are popular therapeutics for a plethora of disease conditions, from cancer ...response. T cell epitopes are found in either the therapeutic antibody itself or as impurities in the formulation... algorithm to evaluate the immunogenicity of therapeutics in silico. These algorithms are trained on vast...algorithms fail to capture. In vitro evaluation of therapeutic immunogenicity is typically performed using traditional...In-vitro models for immunogenicity prediction of therapeutic antibodies. Used under CC BY fromGroell et.al... et.al, 2018). Treating these cells with the therapeutic antibody can help us assess the effects of the...researchers to develop safer and more effective mAb therapeutics. The in silico method of immunogenicity assessment... -
Four Ways to Package Transgenes That Exceed the Size Limit of Adeno-associated Virus
TypeBlog Post...Using rAAV6. Molecular Therapy 19:36–45 . https://doi.org/10.1038/mt.2010.205 Trapani I, Colella P, Sommella...sequence derived from the filamentous phage F1 (Trapani et al., 2014). Drawbacks: The choice of homology...need to be optimized for each unique application (Trapani et al., 2015). Expression of smaller fragments... of Adeno-Associated Virus Capsids. Human Gene Therapy Methods 27:1–12 . https://doi.org/10.1089/hgtb.2015.140...Capacity in a Transgene-independent Manner. Molecular Therapy 16:124–130 . https://doi.org/10.1038/sj.mt.6300322...Reassembly of Adeno-associated Virus. Molecular Therapy 18:6–8 . https://doi.org/10.1038/mt.2009.280 Hirsch...DNA-PKcs-independent, Rad51C-dependent Repair Pathway. Molecular Therapy 21:2205–2216 . https://doi.org/10.1038/mt.2013.184... -
Cancer, Inflammation and Immunity - Harnessing the Body’s Defenses to Fight Cancer
TypeBlog Post...immunology, cancer-immune cell interactions, cancer immunotherapy, as well as new approaches to reprogramming ...reprogramming tumor-associated inflammation for therapeutic benefit ”as stated by the organizers. My overall impression... discussed the effects of starvation on cancer therapy. Findings in previous animal studies suggested ...immunosuppressive reaction in order to improve therapeutic outcomes. If you are studying inflammation and...to help rid us of cancer in combination with chemotherapy. To dive deeper into this field read our recent...doctoral candidate at Binghamton University! Cancer therapeutics The so called field of Immuno-Oncology, a sizzling... hot topic, includes studies on novel cancer therapeutics such as (Chimeric Antigen Receptors) CAR T ... -
Adenoviral Vector Production and Troubleshooting
TypeBlog Post...attractive vectors for research applications and gene therapy: they can be produced at high titers, can accommodate...the Adv genome’s utility in research and its therapeutic potential. Note: constructs discussed are based...Potential applications for gene and cell-based therapies in the new era of personalized medicine (2017)...Gutless AdV have generated high interest for gene therapy due their increased cloning capacity (up to 36 ...20 additional days at which time the cells are scraped, and lysed by multiple freeze-thaw cycles. The ...References 1. Luo, Jinyong, et al. "A protocol for rapid generation of recombinant adenoviruses using the... the AdEasy System. 3. Jager, Lorenz, et al. "A rapid protocol for construction and production of high-capacity... -
Lentiviral Vector Uses and Overview
TypeBlog Post... vectors in cancer immunotherapy. Oldham RA, Berinstein EM, Medin JA. Immunotherapy. 2015;7(3):271-84.... and they’ve also had success in clinical gene therapy applications. Read on to learn more about the current...2nd generation lentiviral packaging system The graphic below shows how the lentiviral genome was condensed...interested in developing these vectors for gene therapy. In 2003, VIRxSYS began the first clinical trial...of 2013, 65 patients had been treated with this therapy, which lowered viral load and did not cause severe...Lentiviral vectors are promising agents for cancer immunotherapy, but this research is still in the pre-clinical...These safer vectors have become standard in gene therapy. Researchers have also developed integrase-deficient... -
Viral Vectors 101: Viral Applications
TypeBlog Post...gene therapy have also been in the limelight. The FDA approved the first oncolytic viral therapy in 2015... science research all the way to clinical gene therapy. But how can you use these amazing tools in your...libraries makes them the ideal vector for the job. Therapeutic applications Vaccine development Attenuated and...being exploited as delivery mechanisms for gene therapy. The tractability of AAV, low immunogenicity, and...for human patients. Multiple viral-based gene therapies have already been approved by the FDA, and this...you are interested in basic science or clinical therapy—viruses could further your efforts. And Addgene... et al. Viral vector platforms within the gene therapy landscape. Sig Transduct Target Ther 6, 53 (2021... -
Viral Vectors 101: Producing Your rAAV
TypeBlog Post...inverted terminal repeat sequences. Human Gene Therapy Methods, 23(1), Article 1. https://doi.org/10.1089... Utilizing minimally purified secreted rAAV for rapid and cost-effective manipulation of gene expression...can limit production of recombinant AAV-2. Gene Therapy, 6(7), Article 7. https://doi.org/10.1038/sj.gt...Inactivation of AAV Serotype 1 Vectors. Human Gene Therapy Methods, 28(1), Article 1. https://doi.org/10.1089...Vectors by Oxidant-Based Disinfectants. Human Gene Therapy, 32(13–14), Article 13–14. https://doi.org/10.1089...genome titers by droplet digital PCR. Human Gene Therapy Methods, 25(2), Article 2. https://doi.org/10.1089...type 2 Reference Standard Material. Human Gene Therapy, 21(10), Article 10. https://doi.org/10.1089/hum... -
Great Results Start with Great Standard Curves
TypeBlog Post...versus the response and use that information to extrapolate the concentration of an unknown sample. For example...standard curve? To create a standard curve, you graph the measured response of the serially diluted standard...absorbance is plotted on the y-axis. With the help of graphing software, you then generate a trendline for the...sigmoidal. Once the trendline is calculated, you can extrapolate the concentration of the unknown sample based...sample is determined by using the trendline to extrapolate its x value, 0.963 mg/mL, from its y value, 0.651...the quality of a standard curve scientists use graphing software to calculate the data’s coefficient of..., manufacturers often recommend using advanced graphing software designed to plot complex trendlines. ...