AnaBios Corporation’s Post

Our PNAS paper is now out - CRISPR gene editing for C9orf72 FTD/ALS in patient iPSC-derived neurons. One step closer to CRISPR therapy for fatal genetic dementia and ALS. https://lnkd.in/gqVDERfM

The November issue of Cytotherapy is out now, and it is loaded with publications from ISCT, International Society for Cell & Gene Therapy's Committees! These four are must reads from leaders in the field of cell and gene therapy translation: Bioprocessing considerations for generation of iPSCs intended for clinical application: perspectives from the ISCT Emerging Regenerative Medicine Technology working group #iPSC #review (https://lnkd.in/gJM2mTad) A new breakthrough in genome editing: the story of Casgevy #GeneTherapy #editorial (https://lnkd.in/ge9mJ4dM) Considerations for the development of iPSC-derived cell therapies: a review of key challenges by the JSRM-ISCT iPSC Committee #iPSC #OpenAccess (https://lnkd.in/eHXRz7e2) International Society for Cell & Gene Therapy Stem Cell Engineering Committee report on the current state of hematopoietic stem and progenitor cell–based genomic therapies and the challenges faced #GeneTherapy (https://lnkd.in/g_GhXh9D) The full Table of Contents can be found here: https://lnkd.in/gtDfSWUP Cover Image: Immunofluorescence staining of F-actin in human limbal mesenchymal stem cells (hLMSCs) Merve Nur Soykan

#aav #machinelearning #genetherapy A team of researchers from the University of California, including University of California, Berkeley and University of California, San Francisco, have developed a machine learning-driven technique to generate varied AAV libraries with strong packaging efficiency. This method involved synthesizing a baseline library, using a predictive model to design optimal libraries balancing diversity and fitness, and validating these designs through experimental measures of packaging success and infectivity in human brain tissues. Additionally, this approach, validated in primary human brain tissue for AAV directed evolution, shows potential for developing targeted gene therapies for brain diseases by improving delivery to specific brain cell types like glial cells. David Schaffer Jennifer Listgarten https://lnkd.in/g6YVZMkM

Joining the trend to highlight exciting studies in the field. This is first of its kind in vivo mRNA-transposase/rAAV-template co-delivery system, demonstrating successful stable genome integration in neonatal mice and NHPs. mRNA-based Sleeping beauty SB100X in conjunction with rAAV based gene correction was stabilized into adulthood following a single delivery to dividing hepatocytes in the newborn liver. In NHPs, integration by transposition mediated by this technology improved gene expression 10-fold. This suggests a dramatic reduction in the required AAV vector dose to achieve clinical efficacy using the hybrid AAV/LNP approach with transposases, potentially increasing safety. Although, authors did not fully address the concerns regarding insertional precision using SB system. https://lnkd.in/eBuKrWce #mRNAtherapy #mRNA

Cell and gene therapies are revolutionizing modern medicine, offering new hope and treatment options for a variety of diseases. Check out this e-book called "Cell and Gene Therapies: From the Lab to the Clinic" that dives into recent advancements, challenges, and clinical applications in this field. From breakthroughs to tackling rare diseases, it's a must-read for anyone interested in the future of healthcare. Download the e-book below 👇 #CellTherapy #GeneTherapy #RareDiseases

This year’s #NobelPrize celebrates #VictorAmbros and #GaryRuvkun for the discovery of microRNAs, a new class of tiny RNA molecules pivotal in gene regulation. Their work sheds light on new horizons in medical research. The breakthrough promises transformative possibilities for personalized medicine. Can't wait to witness how this groundbreaking discovery can impact the landscape of drug development. #NobelPrize #MicroRNAs #MedicalResearch #DrugDevelopment

An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery Developing vehicles that efficiently deliver genes throughout the human central nervous system (CNS) will broaden the range of treatable genetic diseases. We engineered an adeno-associated virus (AAV) capsid, BI-hTFR1, that binds human transferrin receptor (TfR1), a protein expressed on the blood-brain barrier. BI-hTFR1 was actively transported across human brain endothelial cells and, relative to AAV9, provided 40 to 50 times greater reporter expression in the CNS of human TFRC knockin mice. The enhanced tropism was CNS-specific and absent in wild-type mice. When used to deliver GBA1, mutations of which cause Gaucher disease and are linked to Parkinson’s disease, BI-hTFR1 substantially increased brain and cerebrospinal fluid glucocerebrosidase activity compared with AAV9. These findings establish BI-hTFR1 as a potential vector for human CNS gene therapy. https://lnkd.in/egPrBW8e

A team of Australian neuroscientists have identified a new gene therapy target for the neurodegenerative diseases ALS and FTD. They identified a protein interaction partner associated with the cytoplasmic re-localization of pathogenic TDP-43 proteins found in sporadic ALS and FTD. Their results revealed the critical role that 14.3.3ɵ protein plays in the nuclear-cytoplasmic shuttling of TDP-43 and led them to a promising gene therapy slowed or reversed neurodegeneration in ALS/FTD mouse models. Dive into the full findings and explore the implications for ALS and FTD research in our latest blog. https://bit.ly/3PIu2I7

I'm delighted to announce the publication of our latest work in the Genome Research journal. In this study, we achieved three significant milestones: 1) Engineered a groundbreaking endogenous Drosophila tauopathy model through CRISPR-Cas9 gene editing. 2) Utilized cutting-edge scRNA-seq analysis to demonstrate the conservation of tau function across evolutionary time (~600 million years onwards). 3) Uncovered novel mechanisms and potential therapeutic targets for Alzheimer's disease and related dementias. This research substantially advances our understanding of neurodegenerative disorders and offers promising avenues for future therapeutic development. "https://lnkd.in/e6jJi2rs

📢 [NEWSLETTER] 🧬 In this new issue, Frederic Revah reviews the results of gene therapy trial for #Duchenne muscular dystrophy, sponsored by Genethon at Myology Congress and the advances of the limb-girdle muscular dystrophy trial with Atamyo Therapeutics, a Genethon’s spin-off.   🔬 Also discover recent news of our laboratory, including the creation of the GenoTher BioCluster, as Genethon is a founder. The BioCluster is an unique ecosystem in France and in Europe to meet the scientific, technological, and economic challenges related to innovative therapies, particularly #gene therapy.   Do not miss also our activities about research #collaborations and scientific #publications. To read the newsletter 👇 https://urlz.fr/qSxD #genethon #newsletter #advances #dmd #lgmd #biocluster #genetherapy #genother