Publicação de Hilab

Empty versus full - a relevant topic for #AAV characterization 🔬 Are you in need of characterizing your AAVs? One of the most crucial quality attributes of AAV-based products is the genome length determination (or more simply said "empty versus full"). Coriolis Pharma is dealing with this analytical evaluation since 2018, offering different methods to do so. The most promising analytical tools for genome length determination of AAVs are #AUC (analytical ultracentrifugation) and #MP (mass photometry). 🎆 👉 Please approach us if you are in need for analytical support - #RnD or #GMP. https://lnkd.in/dUUcde9h

AlphaFold 3 prevê a estrutura e interações de todas as moléculas da vida. O AlphaFold 3 cria capacidades para o design de medicamentos com previsões para moléculas comumente usadas em medicamentos, como ligantes e anticorpos, que se ligam a proteínas para mudar como elas interagem na saúde e na doença humanas. A previsão experimental da estrutura da proteína pode levar cerca do comprimento de um PhD e custar centenas de milhares de dólares. Nosso modelo anterior, AlphaFold 2, foi usado para prever centenas de milhões de estruturas, o que teria levado centenas de milhões de anos-pesquisadores no ritmo atual da biologia estrutural experimental.

Early AAV developers focus a lot on VG. But that isn't enough.  In fact, only measuring VGs could be a big risk. Here's what you should do instead: First, here's why relying only on vector genomes (VG) can be misleading   -VG ≠ "therapeutically active"   -It’s not a good measure of a preparation quality   -It can easily lead to overestimating batch effectiveness So what should you do? Supplement VGs with functional assays.  They give read-outs as Infectious Units (IU) or Transduction Units (TU). Basically, they tell you how many of your AAV particles actually work. It can often tell a difficult story. For example, unoptimized AAV preps with 10^13 VG/mL. But the same prep can only get 10^11 TU/mL. That's a big difference. So how can you start measuring functionality? Here are two key techniques:   -𝗧𝗖𝗜𝗗𝟱𝟬 𝗮𝘀𝘀𝗮𝘆: Reports infectious units (IU) to assess viral infectivity   -𝗚𝗲𝗻𝗲 𝗧𝗿𝗮𝗻𝘀𝗳𝗲𝗿 𝗔𝘀𝘀𝗮𝘆 (𝗚𝗧𝗔): Using a fluorescent marker, this assay measures transducing units (TU), showing how many vectors successfully deliver their genetic material. So remember... VG can be good for a quick check.  But you shouldn't trust VG alone.  Always double-check function.  Later on, in clinical trials... You'll be glad you did. Anything else you'd add? Drop it in the comments. #genetherapy #aav #analytics #biotechnology 

Mais um passo para desbloquear a investigação contra a leucemia mieloide aguda

Estimular a reflexão sobre o papel emergente das terapias celulares no tratamento das doenças hemato-oncológicas é o objetivo da conferência que a APCL Associação Portuguesa Contra a Leucemia organiza já no próximo dia 23 de março, da qual somos parceiros. Especialistas de várias partes do mundo vão refletir e debater sobre a imunoterapia, partilhando as mais recentes informações nesta área, com destaque para o tratamento com células CAR-T. Uma contribuição relevante para a partilha de conhecimento e experiência entre os profissionais de saúde de diferentes países envolvendo a comunidade de doentes para que possa, de forma informada, fazer parte do processo de decisão e gestão da sua doença.

Introducing the Seegene Inc. Nimbus: A Revolutionary Leap in Multiplex Molecular Diagnostics! At Accuscience, we are thrilled to unveil the Seegene Inc. Nimbus, a state-of-the-art solution designed to streamline and enhance the efficiency of your laboratory workflows. Why settle for less when you can have it all in one robust platform? 🧬 Complete Integration from NA Extraction to PCR Setup Experience seamless laboratory processes with our one-step solution that covers everything from nucleic acid extraction to PCR setup. The Seegene Nimbus ensures that transitioning between different stages of molecular diagnostics is smooth and fully automated. 🔬 All-in-One Platform for Broad Multiplex MDx Assays Tailored to meet the demands of broad multiplexing capabilities, the Seegene Nimbus supports various disease areas with just one platform. Whether it’s infectious diseases, oncology, or genetic disorders, our system handles them all efficiently and effectively. 💡 Cost-Effective Solution Eliminate the need to invest in multiple providers. The Seegene Nimbus offers a comprehensive solution that not only reduces initial investment but also cuts down ongoing operational costs. More savings, better outcomes! 🕒 Maximized Convenience, Minimized Hands-on Time We value your time. That’s why our platform is designed to reduce manual intervention significantly. With selectable functions for either complete automation from NA extraction to PCR setup, extraction only, or PCR setup only, you control the workflow based on your specific needs. 🔍 Enhanced Safety and Accuracy Minimize the risks of contamination and human errors. Our advanced system is engineered to provide a controlled environment that enhances the reliability and integrity of your results. 🧪 High Throughput Capability Scale up your operations effortlessly. The Seegene Nimbus has a capacity to handle up to 72 samples simultaneously, allowing for high throughput and increased productivity in your lab. Transform your diagnostic capabilities today with the Seegene Nimbus. Embrace the future of multiplex molecular diagnostics with AccuScience. Let’s innovate and exceed possibilities together! #AccuScience #MolecularDiagnostics #Innovation #HealthcareTechnology #LaboratoryAutomation #SeegeneNimbus #Pathology #MedicalResearch

#Review CRISPR-Cas-Integrated LAMP by Nazente Atçeken and Savas Tasoglu https://lnkd.in/gywQTbMZ   #MDPI #CRISPR #molecular #Pathogen #biosensors #sensors #openaccess   #Abstract Pathogen-specific point-of-care (PoC) diagnostic tests have become an important need in the fight against infectious diseases and epidemics in recent years. PoC diagnostic tests are designed with the following parameters in mind: rapidity, accuracy, sensitivity, specificity, and ease of use. Molecular techniques are the gold standard for pathogen detection due to their accuracy and specificity. There are various limitations in adapting molecular diagnostic methods to PoC diagnostic tests. Efforts to overcome limitations are focused on the development of integrated molecular diagnostics by utilizing the latest technologies available to create the most successful PoC diagnostic platforms. With this point of view, a new generation technology was developed by combining loop-mediated isothermal amplification (LAMP) technology with clustered regularly interspaced short palindromic repeat (CRISPR)-associated (CRISPR-Cas) technology. This integrated approach benefits from the properties of LAMP technology, namely its high efficiency, short turnaround time, and the lack of need for a complex device. It also makes use of the programmable function of CRISPR-Cas technology and the collateral cleavage activity of certain Cas proteins that allow for convenient reporter detection. Thus, this combined technology enables the development of PoC diagnostic tests with high sensitivity, specificity, and ease of use without the need for complicated devices. In this review, we discuss the advantages and limitations of the CRISPR/Cas combined LAMP technology. We review current limitations to convert CRISPR combined LAMP into pathogen-specific PoC platforms. Furthermore, we point out the need to design more useful PoC platforms using microfabrication technologies by developing strategies that overcome the limitations of this new technology, reduce its complexity, and reduce the risk of contamination.

A Bluebird bio está a assinalar um marco no tratamento da doença falciforme com a primeira utilização comercial da sua terapia genética, Lyfgenia, num rapaz de 12 anos de Washington, DC. O tratamento, autorizado pela FDA em dezembro, é feito a partir das células estaminais do próprio doente, que são carregadas com um gene que codifica uma proteína transportadora de oxigénio defeituosa nas pessoas com a doença. Esta descoberta, levada a cabo por médicos do Children's National Hospital, marca um passo crucial na aplicação comercial desta terapia genética, oferecendo esperança às pessoas afectadas pela doença do sangue. Leia a história completa aqui 👉🏼 https://lnkd.in/dfg3SqKb #tecnologia #medicina #anemoterapia panocítica #terapia genética

The Data and Safety Monitoring Board (DSMB) for the Phase 1/2 ArMaDa clinical trial for OCU410 by Ocugen, Inc. recently convened and approved to proceed dosing with the medium dose of OCU410 in the dose-escalation phase of the study. In the Phase 1/2, 3 patients with geographic atrophy (GA) have been dosed to date. An additional 3 patients will be dosed with the medium dose (Cohort 2) and 3 patients with the high dose (Cohort 3) of OCU410 in the dose-escalation phase. Read More: https://ow.ly/7EGx50RbB37

Publicado na revista Nature Communications Já sabíamos que a CD33 CAR T era uma potencial cart para combater a leucemia mieloide aguda nas crianças mas os resultados não são fantásticos como foram com CD19 CAR T contra a leucemia linfoblastica aguda das criancas. Através da edição do NKG2A com CRISPR/Cas9 da CD33 NK-CAR T cell melhoraram a eficácia da destruição dos blastos in vivo Esta melhoria veio resolver um efeito inibidor dos blastos sobre a interação entre HLA-E e o NKG2A, permitindo que a CAR33-KLRC1ko-NK cells tenham um efeito anti leucemia o elevado tanto in vitro como in vivo. Assim se o NKG2A-deficient CAR-NK cells tem um potencial de ultrapassar a imunosupressao causada pelos blastos da leucemia mieloide aguda. https://lnkd.in/e2Wn8MY9