We narrowed to 431 results for: RAP

...of combining targeted molecular therapies for cancer with chemotherapies, or developing innovative mobile...clinical research to discover and test the best new therapeutics. Consultants often work with such teams to help...human resources towards the development of a new therapy based on its market need and anticipated commercial...context of significant clinical uncertainties and a rapidly evolving healthcare space. The consultants who ...at the core of the suite of benefits a given therapeutic approach brings to patients, doctors, and insurance...and the commercial applications of a particular therapy. With your scientific knowledge behind you, you...business and scientific understanding of successful therapeutics can be a great fit for former healthcare consultants...

... observations: Rapamycin is a membrane-permeable pharmacological agent that can rapidly induction heterodimerization...outer membrane protein with a FRB domain (MitoTRAP). Rapamycin binds your recombinant protein (via FKBP...heterodimerization of proteins that contain rapamycin-binding domains, such as FKBP and FRB. The cell does... the mitochondria (binding FRB), allowing for a rapid and inducible inactivation. Find the Robinson plasmids...approach to control for these effects, drawing on the rapidly growing field of optogenetics. Using a rational... J 2014 Aug 1;33(15):1713-26. RUSH & CRUSH – rapid & conditional gene silencing in RNAi transgenic ...CRUSH alleles off or on enables this method to rapidly address questions of tissue-specificity and cell...

...Power., 2019). Their rapid clearance from blood can be a drawback for therapeutic applications where longer...antibodies (Ahmed et al., 2012, Bates and Power., 2019): rapid blood clearance, which is useful for imaging applications...better tissue penetration which is useful for therapeutic and imaging applications; and reduced immunogenicity... longer retention times often increase therapeutic efficacy (Ahmed et al., 2012). How do you generate ...Just like antibodies, there are many scFv-based therapies in clinical trials or already in the clinic (Bates... for cancer, autoimmune, and/or inflammatory therapeutic applications. Antibody fragments such as scFvs...Monoclonal Antibodies in Immunodiagnosis and Immunotherapy 34:404–417 . https://doi.org/10.1089/mab.2015.0036...

...successfully helped researchers develop a variety of therapies. For example, many scientists use mice to study...the complex interactions between cancer cells, therapeutic drugs, and the rest of the body than studies ...mutations that made cancer cells more resistant to chemotherapy. You can find these plasmids at Addgene. Another...important human disease model with potential for therapeutic drug discovery as it is estimated that 75% of...they can also be used to easily screen multiple therapeutic compounds in one experiment. Zebrafish (Danio...elegans are small, transparent worms that have a rapid life cycle and large brood sizes. The majority of...Drosophila melanogaster and the role of the fly in therapeutic drug discovery." Pharmacological reviews 63.2...

...more hands on While teaching courses about gene therapy and genetic manipulation, I and other professors...similar types of thinking go into developing gene therapy strategies for various diseases. That’s when I ...skills to apply those concepts and understand how therapeutic strategies work. Find Addgene Blog posts about...pick the best strategy and propose a final gene therapy approach for the disease to be openly discussed...impression was that they had clearer ideas about gene therapy in the following lessons. And let me tell you more...they had brilliant ideas on how to solve many therapeutic and pre-clinical problems! Who knows, perhaps...gamma-retrovirus or lentivirus-based ex-vivo gene therapy’ strategies to undergraduates, however you may ...

...lab from the University of Utah developed SapTrap. The SapTrap system uses golden gate assembly to produce...Ras oncogenes have been difficult to target therapeutically due in no small part to the central role Ras...restriction enzyme site and unique primer sites for more rapid screening by restriction digest and Sanger sequencing... sites, this technique also allows for easy and rapid targeting of complex elements to specific genomic...genome engineering method in bacteria that enables rapid, automated, and high-throughput genome editing is...(ssDNA) oligonucleotides (oligos), and thereby rapidly create desired allele combinations and combinatorial...et al. Metab Eng. 2012. PubMed PMID: 2232647   SapTrap, a toolkit for high-throughput CRISPR/Cas9 gene...

...organism. These features make AAV ideal for human therapies and in vivo laboratory experiments ranging from...in the nucleus and is a good option if you need rapid transgene expression, since you don’t have to wait...transduction independently of DNA synthesis." Gene therapy 8.16 (2001). PubMed PMID: 11509958. Vandenberghe...adeno-associated virus manufacturing." Human gene therapy 21.10 (2010): 1251-1257. PubMed PMID: 20649475....Efficient AAV Vector Production System: Towards Gene Therapy For Duchenne Muscular Dystrophy. InTech. doi: 10.5772...the titration of AAV-2 vector stocks."Molecular Therapy 6.2 (2002): 272. PubMed PMID: 12349826. Xiong, ...Various AAV Serotypes and Their Applications in Gene Therapy: An Overview.” Cells, 12(5), 785 (2023). PubMed...

... islandicus CRISPR-Cas system. 5. Phage therapy Phage therapy uses phage to treat bacterial infections...promoters, light, or small molecules could achieve rapid, dynamic, and spatial control of CRISPR-Cas activity...bacterial pathogens possess CRISPR-Cas systems, phage therapy might not be as effective. This is where Acr proteins...enough that they could be engineered to “armor” therapeutic phages to protect them from pathogenic bacteria...activity is an important step towards CRISPR-based therapies and for more precise editing in the lab. References...Toxicity in Human Hematopoietic Stem Cells. Molecular Therapy - Methods & Clinical Development 9:390–401 . https...

...primary human cells. Now, scientists at Beam Therapeutics have found a way to improve editing in primary... and Head of Gene Editing Technology at Beam Therapeutics, used a previously developed bacterial selection...research! Base editing in primary human cells  Cell therapy approaches can use base editors to correct mutations...efficiently makes it a promising tool for T cell therapy development.  Reducing off-target editing in ABE8s...editing specificity, for example for potential therapeutic approaches, the authors recommend mRNA delivery...off-target deamination, an important attribute for therapeutic application. ABE8s demonstrate an overall improved...adenine base editors with increased activity and therapeutic application. Nature Biotechnology. https://doi.org...

...nanobodies that bind to coronavirus spike proteins (Wrapp et al., 2020), which sit on the surface of coronaviruses... read our secondary nanobodies blog post.  A rapid selection platform for generating sybodies against...developed a robust in-vitro protocol that enables rapid selection of synthetic nanobodies, called sybodies...for further characterization and analysis. In a rapid response to the COVID-19 pandemic, the Seeger lab...lab demonstrated they could use the protocol to rapidly identify and isolate sybodies targeting the SARS-CoV...BioRxiv.  https://doi.org/10.1101/2020.04.16.045419.  Wrapp D, De Vlieger D, Corbett KS, Torres GM, Wang N, ...single domain antibodies for the conformational trapping of membrane proteins. eLife 7: . https://doi.org...

... agarose addition, we found it was possible to rapidly punch out cell clones with sterile truncated pipet...from a 10 cm dish in 15- 20 minutes- a far more rapid pace than using cloning rings. A picture of a 60...only warm but not hot- and still liquid- it is rapidly poured onto the middle of the plate, where it immediately...and partially dislodged from the agarose plug by rapid pipetting, as shown in the YouTube video. Before... appear to be yielding well-spaced clones. For rapidly growing cells we sometimes use TPP peelaway T-75...drop of serum to each well in the 48-well plate; rapidly growing cells such as HEK and CHO do not seem to...technique should be useful to scientists who wish to rapidly isolate individual cell clones with different expression...

...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...