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Scientists engineer CRISPR enzymes that evade the immune system By Allessandra DiCorato January 9, 2025 Breadcrumb Home Scientists engineer CRISPR enzymes that evade the immune system The new genome-editing tools could lead to safer, more efficient gene therapies.
What key findings about stem cell behaviour, differentiation and integration within host tissues impact the development of stem cell therapies? Additionally, ADSCs possess immunomodulatory properties, enabling them to modulate immuneresponses and promote tissue healing.
This ebook delves into the intricate world of cancer therapies, immune system breakthroughs, therapeutic genetic engineering, and groundbreaking advancements in gene editing, featuring insightful interviews with renowned experts in the field.
Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain. “As
Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.
To convert this immunosuppressive environment into one amenable to an immuneresponse, investigators engineered a novel oncolytic virus that can infect cancer cells and stimulate an anti-tumor immuneresponse.
NK cells are among the front line of protection from infected and abnormal cells as part of the ‘innate immuneresponse’. They recognise ‘cell stress molecules’ on the surface of infected, old, injured and cancerous cells without the need for complex pre-stimulation signals of the adaptive immune system (eg, T cells).
Researchers have discovered a protein variant that serves as a knob for regulating the body's innate immuneresponse. The findings could lead to new therapies for Long COVID, autoimmune disorders, and more.
In the realm of immunology, researchers are constantly striving to unlock the intricate secrets of the human immune system. Among the many remarkable components that make up this complex defence network, T cells have emerged as powerful guardians, orchestrating an array of immuneresponses with great precision.
Here, we have developed a targeted photosensitizer for photothermal therapy (PTT) of solid tumors through the functionalization of single walled carbon nanotubes (SWCNTs) to ANXA5-the SWCNT-ANXA5 conjugate. In vitro ablation of cells incubated with the conjugate promoted cell death in a dose-dependent and targeted manner.
Researchers have demonstrated that a transplant surgical procedure (called 'needle trauma') triggers a profound immuneresponse and causes the death of most grafted dopamine neurons. These findings suggest a path for the 'realistic' use of cell therapy to treat neurodegenerative disorders.
Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.
NK cells are among the front line of protection from infected and abnormal cells as part of the ‘innate immuneresponse’. They recognise ‘cell stress molecules’ on the surface of infected, old, injured and cancerous cells without the need for complex pre-stimulation signals of the adaptive immune system (eg, T cells).
How significant are the results in terms of advancing TCR-T cell therapy and AMP immunotherapy for the treatment of solid tumours? We have already applied this approach to several important tumour targets where enhancements in TCR-T therapy may lead to important gains in clinical benefit, including mutated KRAS, NY-ESO, and HPV E7 antigens.
doi: 10.1038/s41590-024-01792-2 (2024) Scientists used a microscope to visualize an immunity hub (yellow and green) in human lung tumor tissue (blue). Networks of cells are critical in mediating immuneresponses. How do immune cells organize within tumors to effectively eliminate cancer cells? Nature Immunology.
Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.
Cell-based therapies, such as tumour-infiltrating lymphocyte (TIL) therapy, have also drawn significant attention following promising results in a melanoma trial. Such approaches apply artificial intelligence (AI) and machine learning (ML) to predict mutations most likely to be good targets for the immune system.
2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. These can include anti-tubulin compounds, potentially reducing side effects and improving quality of life.
In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.
Repurposing of approved therapies is the fastest way to impact patients today, as these medicines have regulatory approval to enable investigator-initiated trials and have a manufacturing process to ensure drug supply. (You can download a pdf copy of the blog here.) appeared first on Plenge Gen @rplenge.
Better activation of innate and adaptive immuneresponses was achieved with CV2CoV, resulting in faster response onset, higher titers of antibodies, and stronger memory B and T cell activation as compared to the first-generation candidate, CVnCoV. “In Induction of innate immunity was investigated via specific cytokine markers.
Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.
Terpenes are compounds that inhibit the growth of Leishmania spp, Schistosoma spp, and Echinococcus spp, cause changes in parasite ultrastructure, inhibit enzyme activity, and modulate the immuneresponse. Their use in combination therapy decreases the concentration of the reference drug used.
In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. These neoantigens are identified by T cells of the immune system as foreign proteins and thus trigger an immuneresponse. Neoantigens are recognised as non-self and trigger an immuneresponse.
His mother had a presentation of the disease that suggested her immune system was already on the job. But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way.
Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Stem cell transplants primarily help the immuneresponse through the “graft-versus-leukaemia effect,” and we have to manage the “graft versus host effect.”
There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. AAV-based therapies often can’t be dosed more than once due to concerns about an immuneresponse. Molecular Therapy-Nucleic Acids. Human molecular genetics.
In research published in Scientific Reports , 1 investigators focused on mesenchymal stem cells (MSCs), known for their potential in treating cell defects and regulating immuneresponses. We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun.
Targeting 4-1BB remained of interest to the immuno-oncology field as cell culture experiments and tumor models in mice suggested that robust anti-tumor immuneresponses could be triggered by anti-4-1BB antibody therapies. This is the T cell type most closely associated with anti-tumor immuneresponses.
More recently, researchers have begun to look at conjugating immunomodulatory agents to antibodies to directly activate an immuneresponse against the tumour. This approach – immune-stimulating antibody conjugates (ISACs) – uses a payload that stimulates the innate and adaptive immuneresponses, recruiting tumour-fighting T cells.
My Attendance at the 2024 Boston Society Cell and Gene Therapy Conference pmjackson Tue, 06/11/2024 - 20:49 , via Wikimedia Commons" data-entity-type="file" data-entity-uuid="c7a7fa8b-b2fe-4d84-a75e-d1ba3a4e2caf" src="[link] width="742" height="249" loading="lazy" /> Ecm85, CC BY-SA 3.0
We are in an era of immuno-oncology (IO) revolution with many approved therapies now available to treat a broad range of cancers. Generally, IO has been focused on harnessing the anti-tumour activity of certain cancer-fighting T-cells , a key cell type involved in the adaptive immune defense system.
The preclinical results demonstrated significant immuneresponses and tumour regression. Could you elaborate on the mechanisms by which these responses are initiated within cancer cells? This induced anti-tumour immunity is also detectable by increased innate and adaptive immune cells in the tumour, spleen and blood.
Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.
They discovered that these same nerve cells responsible for triggering the itch sensation also played a critical role in preventing an overactive immuneresponse and maintaining inflammation at manageable levels. In the absence of IL-31, these nerve cells failed to control the immune system, leading to unchecked inflammation.
HLA are the critical molecules that our immune systems use to present peptides to T cells and facilitate recognition and killing in immuneresponses to pathogens. HLA-II presents peptides to CD4 T cells thought to be important for indirectly helping immuneresponses and facilitating antibody production.
This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.
This is particularly pertinent in immune-based cell therapies like CAR-T, where animals do not mimic the human immuneresponse. The full potential of PDOs would be to shorten the time and costs associated with medicine creation, and then use PDO therapies to treat patients effectively.
Understanding how proteins operate in cancer cells raises the prospect of new therapies that block key proteins that drive cancer growth, or therapies that trigger immuneresponses to abnormal proteins created […]
Use of AAVnerGene capsid library provided to Neurophth for ophthalmic gene therapy.
Next Generation AAVs enhance gene therapies by increasing transduction efficiency and specificity while reducing immuneresponses and cost.
gene therapy company (hereafter Neurophth), and AAVnerGene Inc. ,
Confo Therapeutics , led by CEO Dr Cedric Ververken, is at the forefront of developing innovative GPCR-targeted therapies using its proprietary ConfoBody ® platform. The MC4R-based ConfoGen was used to immunize llamas, resulting in an immuneresponse that generated a highly diverse panel of MC4R-specific agonists.
These connections provide all-too-important ‘reasons to believe’ for further investment and movement into earlier lines of therapy. However, despite this long winter, new approaches, ranging from CD3 bi-specifics, to cell therapies, to cancer vaccines, and more recent novel intracellular mechanisms have started to turn the tide.
This interaction inhibits inflammation and immune activation, and in particular prevents macrophages from phagocytosing and destroying cancer cells. By blocking CD24 with an antibody drug, our goal is to powerfully reactivate the anti-cancer immuneresponse and drive therapeutic efficacy.
Antibodies protect us by attaching to molecules on those invaders (known as antigens) and triggering our body’s natural immuneresponse to destroy them. This can create an abnormal immuneresponse that attacks the cells of our bodies and contributes to the development of autoantibody diseases.
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