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New ENaC blocker demonstrates an extended duration of action
The novel drug, ETD001, could provide an improved approach for mucus clearance in cystic fibrosis patients.
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Cell & gene therapy
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Macrophage-mediated phagocytosis: treating solid tumours
A new immunotherapy approach combining co-engineered T cells and antibodies enhances phagocytosis of tumour cells.
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The promise of vectors targeting the LDL receptor
In this Q&A, Pascaline Lécorché, Head of Chemical Biology at Vect-Horus, outlines the potential of RNA interference therapeutics and the challenges of their use in vivo. There are several strategies to overcome these, and why peptide-based vectors targeting the LDL receptor are a promising approach.
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Novel version of CRISPR gene-editing protein engineered
Researchers have developed a new, compact EbCas12a variant that can be packaged into an all-in-one AAV system with its crRNA.
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New method to predict effectiveness of MSCs for cartilage repair in vitro
Topological defects in mesenchymal stromal cell self-assembly could suggest their potential for cartilage regeneration.
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Enhancing gene therapy with Circio
In this Q&A, Erik Wiklund, CEO of Circio, explains the key findings of their circVec circular RNA platform technology, why they chose AAV-based gene therapy for AATD as the lead programme, and their plans for the future to enhance the potency and reduce the cost of current gold-standard gene therapy.
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Gene editing for HSV-1 shows significant reduction of viral load
The experimental therapy eliminated 90 percent of HSV-1 after facial infection and 97 percent of HSV-1 after genital infection.
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Beyond the lab: cell and gene therapy
Download this report now to access exclusive content on innovative cancer treatments, regenerative medicine, gene therapy, and expert insights from leading professionals in the field.
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New findings could influence asthma drug development
Researchers have revealed that genetically enhanced expression of GSDMB causes a disturbed interferon-response.
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FOXO1 improves survival and function of CAR T cells
Evidence indicating that FOXO1 plays a unique role in promoting T cell longevity could result in more effective CAR T cell therapies.
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Optimising CRISPR gene editing of hard-to-transfect cells
CRISPR has transformed gene editing, but still presents challenges in hard-to-transfect cells, such as pluripotent stem cells and primary cells.1 The key to obtaining successful transfection in these cells lies in innovative workflows. Here Georges Müller, CEO and cofounder of SEED Biosciences, shares his perspective on why focusing on editing…
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Investigating new treatments for polycystic kidney disease
Organoid models enabled the researchers to study the effectiveness of eukaryotic ribosomal selective glycoside drugs on PKD cyst formation.
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New strategies to overcome CAR T-cell resistance in AML cells
Pharmacological concomitant therapies or further genetic improvement of CAR T-cells can increase their effectiveness against TP53-mutant AML cells.
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Developing an mRNA therapy for Duchenne muscular dystrophy
In this Q&A, Aki Ko, CEO and co-founder of Elixirgen Therapeutics, elucidates how their new mRNA technology could potentially restore muscle function in those suffering from Duchenne muscular dystrophy.
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Evaluating novel gene therapies in a whole human liver
Use of the whole liver could revolutionise the development of viral vectors, providing more effective treatments for inherited diseases.