John Gordon’s Post

Here is an update from our first funded Graeme McDaniel Research Scholar, Dr. Turaga, from Texas Children's Hospital, regarding his work, Single nucleus transcriptome of a “Super RV” shows increased insulin and angiogenesis signaling. In certain serious conditions like congenital heart disease, the RV may pump blood into the body instead of just to the lungs. In very rare cases, some people have what's called a 'super RV', where these stressed RVs continue to function well even into later stages of life. In this study, we looked at the genetic activity of ‘super RV’ from a 60-year-old person who had a specific type of congenital heart defect called congenitally corrected transposition of the great arteries (ccTGA). We found that in this person's RV, there were two important pathways that were more active than usual, a pathway related to insulin-like growth factor (IGF1) was more active and increased activity in pathways related to the growth of new blood vessels (angiogenesis). These findings suggest that in rare cases, the RV might last much longer by adapting to the extra strain by becoming more responsive to growth signals and by growing new blood vessels. Understanding how the RV adapts to increased stress is important for developing better treatments for congenital heart disease. Now that we know 'WHAT' genes to target, next steps will be 'HOW' to target the genes in the heart. We can potentially repurpose existing drugs which target these pathways (used for cancer, endocrine diseases) to treat RV failure or use gene therapies to specifically treat heart cells. Both approaches are promising, now that we know 'WHAT' to target. The detailed paper can be found here: https://lnkd.in/gD_WB4ZV Thank you for your dedication to the field, Dr. Turaga! If you are a researcher and your work aligns with our mission, you can find our grant inquiry form here: https://lnkd.in/gS2xNiDx We will fund our 3rd round of grants this summer! #ResearchUpdate #graememcdanielfoundation #congenitalheartdefects #1in100babies #chdresearch #chdawareness

Family Matters: Testing for Inherited Gene Mutations Cancer is a genomic disease in that gene alterations drive the cellular growth and immune surveillance perturbations that enable malignant cells to take hold and to metastasize. The incorporation of next-generation sequencing (NGS) in routine clinical practice has led to the identification of cancer-predisposing mutations in an increasing proportion of patients with several types of cancers. Read more on our blog: https://lnkd.in/diWRTKM9

CRISPR-based therapies hold incredible promise but face challenges with immunogenicity. Pre-existing immunity to Cas proteins, which has been reported in healthy adults, is a key concern for in vivo CRISPR trials. Our new PEPperCHIP® CRISPR/Cas Immunogenicity Microarray can be used for: 👉 Screening of pre-existing antibodies against Cas proteins 👉 Identification of antibody profiles linked to treatment efficacy or adverse effects 👉 Optimization of gene therapy protocols for safer, more effective CRISPR therapies 👉 Immune response monitoring of patients receiving CRISPR-based therapies 👉 Comparing the immunogenicity of different Cas9 variants Learn more about our newest microarray here: https://lnkd.in/ednfqD9Y #CRISPR #GeneTherapy #Immunogenicity

🔬✨ New Protocol Alert! Protocol to establish an accelerated murine model for Helicobacter-induced gastric cancer by Bali et al. from Marygorret Obonyo’s lab from UC San Diego   Summary: Learn how to establish an accelerated murine model for Helicobacter-induced gastric cancer, featuring rapid disease progression with precancerous lesions developing within 6 months post-infection. Protocol Highlights: Explore the accelerated model for Helicobacter-induced gastric cancer, covering infection procedures, disease severity assessment, and gene expression analysis techniques for studying disease progression. Read the full protocol here: https://lnkd.in/e2gn5xkD #STARProtocols #GastricCancerModel #HelicobacterInfection #ResearchMethods #Protocols #CellPress

CRISPR-based therapies hold incredible promise but face challenges with immunogenicity. Pre-existing immunity to Cas proteins, which has been reported in healthy adults, is a key concern for in vivo CRISPR trials. Our new PEPperCHIP® CRISPR/Cas Immunogenicity Microarray can be used for: 👉 Screening of pre-existing antibodies against Cas proteins 👉 Identification of antibody profiles linked to treatment efficacy or adverse effects 👉 Optimization of gene therapy protocols for safer, more effective CRISPR therapies 👉 Immune response monitoring of patients receiving CRISPR-based therapies 👉 Comparing the immunogenicity of different Cas9 variants Learn more about our newest microarray here: https://lnkd.in/ejAip5RP #CRISPR #GeneTherapy #Immunogenicity

Sad news from a gene therapy trial aimed at treating young boys suffering from the rare and fatal genetic disease cerebral adrenoleukodystrophy (ALD). Although most of the boys showed no major disabilities an average of six years after receiving lentivirus-modified (Skysona) blood stem cells, the functional copy of the ABCD1 gene delivered during treatment was found integrated into or near cancer-related genes, as well as other genes that together may have driven uncontrolled blood cell growth. So far, 10% of the boys in the Skysona trials have developed leukemia. While lentiviral vectors have generally been considered safer than other genome-integrating retrovirus vectors, there had been some signs of concern prior to these new findings, now published in The New England Journal of Medicine (NEJM). A similar lentiviral vector has been used in other gene therapies without causing cancer, and the reasons for this discrepancy are currently under investigation. #Lentivirus #Gene_Therapy #Adverse_Effect #Cancer #Leukemia #Skysona https://lnkd.in/dFS5W7nW

CAR-T and antibody therapy are two leading treatments for cancer. How do they compare to each other? Let's break it down. CAR-T - provides long-lasting immune responses - for some blood cancers, CAR-T have shown potential to completely cure the disease - where other options failed HOWEVER - They are more difficult to produce in large quantities (compared to antibody production) - In the tumor environment, they struggle to remain effective Therapeutic antibodies - Much easier to produce in large quantities - Some antibodies can interfere with receptors related to cell growth and blood vessel growth in tumors HOWEVER - Often, repeated administration is required (although some strategies are being developed to potentially fix that) - They often require other immune system components to be activated for their full effect to be appreciated I know I have them "competing" against each other here... But I'm excited to see combination therapies of these two treatments. For a curated list of content that simplifies the science of cell and gene therapy, check out #cgt_simplified Got any additional context to add here? Drop it in the comments. “Created with BioRender.com” #celltherapy #biotechnology #cartcelltherapy #antibodies #science

A new study has found an interesting link between gene mutations in blood #cancer and #arthritis. The publication explains how specific gene mutations in blood cancer can impact autoimmune diseases such as seronegative RA. The study suggests that effective metabolic therapies in treating blood cancer may help arthritis patients.   → Conclusion: • IDH mutant myeloid neoplasms are associated with seronegative rheumatoid arthritis. • High levels of 2-hydroxyglutarate mediate IDH-associated activation of innate immune response.   → Lih En Hong, Mihir D Wechalekar, and Devendra Hiwase led the research team at the Royal Adelaide Hospital in Australia for this study.    → Publication: Hong, Lih En, et al. "IDH Mutant Myeloid Neoplasms are Associated with Seronegative Rheumatoid Arthritis and Innate Immune Activation." Blood (2024).   📖 Read on…https://lnkd.in/egV5am-P   #Inflammation #Immunotherapy #DrugDevelopment

Snapshot of #cancerimmunotherapy with #oncolyticviruses. Oncolytic viruses (OVs) preferentially infect and kill cancer cells without harming normal cells. OVs can revert cancer-associated immune suppression and initiate clinically meaningful antitumor immune responses. OVs and their resultant immunological events can act at both primary and metastatic sites. Thus, OVs can be exploited for cancer gene therapies and immunotherapies alone or in combination with other interventions, including immune checkpoint blockade. Find out how YXgene can help your research: https://lnkd.in/ek-92bbg #immunology #immunotherapy #cancertherapy #genetherapy #celltherapy Image from https://lnkd.in/eaqvVdaT

Leverage MLM's PBMC expertise to drive successful preclinical and clinical trial outcomes, particularly in the fields of Immunotherapy, Immuno-oncology, Cell and Gene Therapy, Infectious Disease, Autoimmune and Inflammatory Disease, Vaccine Development, and Regenerative Medicine. LEARN MORE >> https://lnkd.in/gGfV7ECU #Immunotherapy, #ImmunoOncology, #CellAndGeneTherapy, #InfectiousDisease, #AutoimmuneDisease, #VaccineDevelopment, and #RegenerativeMedicine