We narrowed to 503 results for: RAP

...commonly encountered in human gene therapy trials. Human Gene Therapy Methods, 26(2), 71–76. https://doi.org...dose–response and comparison to AAV PHP.B. Gene Therapy, 25(5), 392–400. https://doi.org/10.1038/s41434... Gene Delivery to the Hypothalamus. Human Gene Therapy, 21(6), 673–682. https://doi.org/10.1089/hum.2009.169...Virus Serotype 9 (AAV9) Preparations. Human Gene Therapy Methods, 30(6), 214–225. https://doi.org/10.1089...Chemically and Functionally Distinct Vectors. Molecular Therapy - Methods and Clinical Development, 18(September...vector purification and formulation. Molecular Therapy, 12(1), 171–178. https://doi.org/10.1016/j.ymthe...

...of the VGS process and use cases in Human Gene Therapy. Existing methods for AAV quality control Historically...Single-stranded Virus Next-generation Sequencing. Molecular Therapy - Nucleic Acids 4:e260 . https://doi.org/10.1038..., Paulk NK (2019) Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded...Viral Genomes in Academic Settings. Human Gene Therapy Methods 30:195–205 . https://doi.org/10.1089/hgtb...Moullier P, Ayuso E, Vandenberghe LH (2017) AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency ...Consistency Evaluation of AAV Preparations. Molecular Therapy 25:1375–1386 . https://doi.org/10.1016/j.ymthe.2017.04.001...

...Wanisch  Listen to the optogenetics podcast segment LOVTRAP is an optogenetic system that was recently developed...promoting domain. Each half was fused with one of the LOVTRAP domains. When both halves of the modified EB1 protein...system: A versatile in vivo fluorescent reporter for rapid quantification of base-editing and Cas9 cleavage...popularity of single base-editors has resulted in the rapid expansion of the genome editing toolkit. However...technologies across several cell types is the lack of a rapid and quantitative editing assay. To help accelerate...has deposited a reporter plasmid that allows for rapid fluorescence-based quantification of base editors...

... antibodies. This is particularly useful for therapeutic applications in vivo where endogenous anti-AAV...anti-AAV antibodies often neutralize the therapeutic efficacy of AAV vectors. How are exosome AAVs produced...interest special contexts, such as the delivery of therapeutic proteins to the brain which requires crossing...cells using exosome-associated AAV." Molecular Therapy 25.2 (2017): 379-391. PubMed PMID: 28082074. PubMed...vector as a novel gene delivery system." Molecular Therapy 20.5 (2012): 960-971. PubMed PMID: 22314290. PubMed...Overexpression of the Tetraspanin CD9." Molecular Therapy-Methods & Clinical Development 9 (2018): 278-287...

...retro-orbital) to intermediate (tail vein) Intraperitoneal Intraperitoneal (IP) injections are given in the peritoneal...drugs and viral vectors. Like IV injections, intraperitoneal injections require a little bit of practice...purposes, but mainly for the delivery of various therapies and viral vectors. This injection sounds very ... that require ingestion, such as experimental therapies or pain relief, straight to the stomach. This ...retro-orbital) to intermediate (tail vein) Intraperitoneal Scruffing Peritoneal cavity 25–27 < 2–3 mL ...

...used for many years for in vivo delivery and gene therapy and represent one of the most studied viruses. ...and very efficient introduction of DNA into therapeutically relevant, non-transformed mammalian cells. ...deficiency (most widely used so far) or for cancer gene therapy such as nonalcoholic steatohepatitis cancer. In...Targeting. In: Applications of viruses for cancer therapy. Elsevier, pp 39–67 Ding Q, Strong A, Patel KM,... Zamyatnin AA Jr (2016) Viral vectors for gene therapy: Current state and clinical perspectives. Biochemistry... of Cas9-Specific Immune Responses. Human Gene Therapy 26:432–442 . https://doi.org/10.1089/hum.2015.087...

...over two decades, attempts to answer it in five paragraphs, with examples. If that sounds ambitious, that...Science” described in Chapter 3, and of course the rapidly changing world of genome engineering via CRISPR...implications of using these technologies, but also the rapidity of the technologies’ adoption by the scientific... also spends some time on Addgene’s role in the rapid dissemination of these technologies, proposing that...are reading this review--in the current period of rapid flux, the chapter might still feel slightly dated...them wanting to learn more. There are individual paragraphs that may have warranted their own chapters, and...

...test for mouse iPSCs, tetraploid complementation, involves injecting tetraploid blastocysts with iPSCs...this approach is still not considered safe for therapeutic applications. piggyBac (PB) transposon: The PB...safer way to express reprogramming factors for therapeutic applications. Sendai viral vectors: Sendai virus...is considered a safer approach for developing therapeutic applications, but it can take a long time to ...induced pluripotent stem cells in research and therapy." Nature 481, no. 7381 (2012): 295-305. PubMed ... cell generation. Expert opinion on biological therapy, 10 2, 231-42. PubMed PMID: 20088717. PubMed Central...

...CRISPR/Cas based COVID-19 testing methods.   Rapid detection of 2019 novel coronavirus SARS-CoV-2 using...incorporated into a lateral-flow readout slip that allows rapid detection.  Highlights: Cas13a is not catalytically...All-in-one dual CRISPR/Cas12a (AIOD-CRISPR) assay – for rapid, ultrasensitive and visual detection of novel coronavirus...operated, made accessible by the DIY-Bio movement. Rapid, field-deployable nucleobase detection and identification... D., Sharma, N., Kumar, M., … Maiti, S. (2020). Rapid, field-deployable nucleobase detection and identification..., & Alejandra, G. C. (2020). An ultrasensitive, rapid, and portable coronavirus SARS-CoV-2 sequence detection...

...diagnostic tools and therapeutics. In addition, the strategic use of whole phages as therapeutics, which is also...long-term library storage. After overnight growth, scrape up, pool and extract plasmid DNA directly from ...Nobrega, Franklin L., et al. "Revisiting phage therapy: new applications for old resources." Trends in...drug approval pathways for bacteriophage-based therapeutics."Frontiers in Microbiology 7 (2016). PubMed ...PMCID: PMC4728577. 4. Schmitz, Jonathan E., et al. "Rapid DNA library construction for functional genomic ...

... in therapy. The lungs are difficult, but other tissues like the retina have cell-based therapies underway...researcher. Darrell's ultimate goal - to develop therapies to treat disease in the lung. Chapter 2: Balancing...disease through tissue repair, stem cells, gene therapy, and tissue engineering. How the multidisciplinary...Resources from Addgene Learn about Retinal Gene Therapy from Connie Cepko Find Plasmids from the Kotton...

...genome engineering - such as certain forms of gene therapy - avoiding the use of viruses is also important...of each construct. This new tool kit allows the rapid generation of stable transgenic cell lines which... CD19-directed chimeric antigen receptor (CAR) therapy. SB is a valuable tool and an alternative to CRISPRs..., et al. Optimized Sleeping Beauty transposons rapidly generate stable transgenic cell lines. Biotechnol...PubMed. 3. Singh H, et al. A new approach to gene therapy using Sleeping Beauty to genetically modify clinical-grade...

...of text. Since our guides are heavily used, our graphic designer made most of the Antibody Guide figures...communication between our content team and our graphic designer to make sure things are clear, accurate...Fig. 2: Reference image (left) provided to our graphic designer upon request that the science communicator...design (right). Immediate and immense regret of the graphic designer not shown.   However, for our blog...retiring. CRISPR/Cas9, for example, has gone through rapid change in the past few years. Thus, we are constantly...

...outlined below as well. Cas12a also has significant therapeutic promise due to the fact that it is smaller than...simplicity of Cas12a makes it easier to package for therapeutic delivery by AAV, which has strict capacity limitations...sequences of Cas12a to further optimize it for therapeutic and laboratory usage. Fast facts – PAM requirement...TTTN. Best for: homology-directed repair or therapeutic delivery.   Cas14 – Mini, but single strand ...stirred up interest for its potential as a viral therapeutic that could specifically degrade target viral ...

...analysis of the US Women’s Health Initiative Hormone Therapy Trial, for example, found that the study saved ...Reduced use of estrogen plus progestin hormone therapy resulted in 76,000 fewer cases of cardiovascular... cells and tissues, specifically in stem cell therapies. Let’s go back to why ten drugs were withdrawn... shows that there are sex differences in the therapeutic capacity of stem cells. Yet, very few researchers... both sexes ensures that the diagnostics and therapeutic interventions you develop represent the whole...

...your study is aimed at developing human therapies. If a therapeutic is the end goal, it is best to work with...Targeting is also important in the development of therapies when expression must be restricted to specific...Varenicline has been further modified to create ultrapotent PSEMS (uPSEMs) that have drastically increased... A, Basu J, Michaelides M, Sternson S (2019) Ultrapotent chemogenetics for research and potential clinical...

...vectors: The Rubik’s cube of human gene therapy. Molecular Therapy, 30(12), 3515–3541. https://doi.org/10.1016... vector for gene therapy of human diseases. Signal Transduction and Targeted Therapy, 9(1). https://doi.org... liver, interrogate immune cells to improve immunotherapy in glioblastoma, and generate programmable screening...

... that CRISPR technology is consistently showing rapid development and quick adoption by the community....request and use new tools. Because there’s such rapid adoption, we thought that requests would be biased...CRISPR tools that fill needs in the community. The rapid adoption of new tools, illustrated in Figure 2, ...usefulness of popular CRISPR tools is both enduring and rapidly recognized.   Where is the CRISPR community?  Addgene...looked at depositors and requestors by country, and graphed the top ten countries for each group (Figure 4)...

...mutations do arise and cancer ensues, traditional chemotherapies have taken advantage of the universally-dividing...Further, depending on the specific type of cancer, chemotherapy is not always effective. This is why recent ... They have proven very difficult to target therapeutically due to their unusually “smooth” shape and lack... high-affinity binding targets for potential therapeutic agents. Nevertheless, a glimmer of light remains...pathways. The curation project continues to evolve rapidly and, with assistance from the research community...

... Adenovirus Vectors for Gene Therapy, Vaccination and Cancer Gene Therapy. Curr Gene Ther, 13(6), 421–...was first isolated from human adenoid tissue and rapidly caused the death of adenoid cells in vitro (Rowe...oncolytic virus in addition to standard uses in gene therapy and in basic research, like other viral vectors...novo viral gene expression: Implications for gene therapy. Proc Natl Acad Sci U S A, 95(19), 11377. https...