Dianox’s Post

Excited to share my latest article on gene therapy and intramuscular plasmid DNA delivery via PEFs (pulsed electric fields)! 🧬 This study delves into the complex world of STING signaling and palmitoylation in mouse skeletal muscle. Findings suggest a unique relationship between STING activation and PEFs. Read the full paper for a glimpse into the molecular intricacies of PEF application. #GeneTherapy #Research #SciencePublication

What separates ResolveDNA WGA from other whole genome amplification kits out there? Hear from Aleksei Mikhalchenko, PhD, Genome Informatics Lead at the Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University ⤵   "Our research depends critically on the ability to sequence single-cell DNA and accurately identify its genomic variants. Among all the whole-genome amplification (WGA) methods we have tested, PTA-based WGA has provided the most accurate output. It reliably stays close to the original 50/50 ratio of allele frequency in heterozygous loci, minimizing allele dropout and false-positive loss of heterozygosity. This makes it an invaluable tool for our research." Don’t settle for whole genome amplification kits that make you choose between high fidelity, even, or complete coverage. Our novel PTA-powered ResolveDNA WGA kits provide all three. Learn more: https://lnkd.in/gm37WwEJ

AAV genome truncations present a significant challenge in gene therapy development, reducing production lot yields and driving up manufacturing costs. The ability to reliably and reproducibly predict the propensity of vector designs for producing truncated genomes early in discovery can help mitigate these manufacturing issues later, saving significant time and cost. In this case study, we investigate whether AI can reliably and reproducibly predict AAV genome truncation propensity and validate the predictions with long-read sequencing data. Explore the methods and results: https://hubs.li/Q02LwD350 #genetherapy #AI #AAV #biomanufacturing

The Hormel Institute hosted the Viral Manufacturing and Translational Consortium (VMTC), bringing together experts from around the world to discuss one of the most exciting frontiers in medicine: viral vector production for genetic therapies. A key highlight was hearing from David Largaespada, our Co-Director at the Center for Genome Engineering. As Dr. Largaespada emphasized, collaboration between academia, industry, and regulatory bodies is crucial to making these therapies available to all who need them. Events like VMTC show just how close we are to achieving these transformative breakthroughs. Their work is helping address the biggest bottleneck in gene therapy: streamlining viral vector production to lower costs and accelerate delivery to patients. 🔬 Big thanks to all the incredible speakers and researchers driving innovation forward. Together, we’re shaping the future of medicine! The Hormel Institute, University of Minnesota University of Minnesota Medical School #GeneTherapy #ViralVectors #GenomeEngineering #VMTC2024 #HormelInstitute https://lnkd.in/gfmAPZbv

Familiarize yourself with the basics of gene therapy and best practices for patient care in this course from ACMG - American College of Medical Genetics and Genomics. #genetherapy https://lnkd.in/gViKfSib

Lentivirus (LV) is the leading vector for ex vivo gene therapy (GT) as it can efficiently transduce different blood cells. As the GT field is now expanding from rare genetic disorders to diseases affecting larger patient populations, there is an increasing need for scalable and affordable LV production systems. This paper describes the development of stable suspension producer cell clones for scalable and serum-free LV production. The cells were capable of maintaining a productivity of over 1 × 10+7 TU/mL/day for up to 29 consecutive days in a perfusion-mode stirred-tank bioreactor, supporting a fully continuous manufacturing process development. Contains also good discussion of, and comparison to, other advanced LV production methods.   #GeneTherapy #LV #Suspension #SerumFree #Production https://lnkd.in/dyH9uzPM

#ARVO2024 join us for a lighting session tomorrow on how I developed multiple therapies to treat rare and common retinal diseases! And learn how #ShifaPrecision will use precision medicine to identify the most effective personalized therapies!

There's been some important research into treating MND, and we've made it more readable. A study found using a gene therapy drug to act like a vacuum cleaner could remove a harmful protein from nerve cells, stopping progression of disease even at very advanced stages. So far, scientists have only tested this in cells and in animals, but funding for human trials has now been secured. Read the summary from Debarati Bhattacharya at https://lnkd.in/er3skdPn #mnd #mndresearch #scientificresearch #sitran