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As the industry looks beyond CRISPR to safely introduce therapeutic genomic changes anywhere in the body, in vivo gene editing holds immense potential to address diseases with a genetic basis. Boston-based biotech Tessera Therapeutics is pioneering the next generation of genetic medicines with its Gene Writing™ platform. At this year’s…
The novel drug, ETD001, could provide an improved approach for mucus clearance in cystic fibrosis patients.
A new immunotherapy approach combining co-engineered T cells and antibodies enhances phagocytosis of tumour cells.
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.
Researchers have developed a new, compact EbCas12a variant that can be packaged into an all-in-one AAV system with its crRNA.
Topological defects in mesenchymal stromal cell self-assembly could suggest their potential for cartilage regeneration.
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.
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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Researchers have revealed that genetically enhanced expression of GSDMB causes a disturbed interferon-response.
Evidence indicating that FOXO1 plays a unique role in promoting T cell longevity could result in more effective CAR T cell therapies.
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…
Organoid models enabled the researchers to study the effectiveness of eukaryotic ribosomal selective glycoside drugs on PKD cyst formation.
Pharmacological concomitant therapies or further genetic improvement of CAR T-cells can increase their effectiveness against TP53-mutant AML cells.
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.
