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Brain organoids: investigating the progression of Huntington’s disease
Researchers found that HD can impair early brain development through defects associated with mitochondrial dysfunction.
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Genome Editing
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Reprogramming B cells to produce custom antibodies
The new technique could be adapted to produce a wide range of antibodies to combat chronic conditions like HIV.
news
New model offers a unique method to study Parkinson’s disease
Mice with rod-specific VPS35 deletion demonstrate a pathology more similar to human Parkinson’s disease, compared to other mouse models.
news
Prime editing corrects the CFTR gene mutation
The approach precisely and durably corrects the CFTR mutation in human lung cells, which could lead to superior treatments.
news
Cancer dependencies missed by some CRISPR guides
Scientists discovered that, depending on germline variation, CRISPR-based experiments can result in false negatives.
article
Tessera Therapeutics: addressing Alpha-1 Antitrypsin Deficiency
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…
news
New biological pathway in IBD discovered
Researchers have identified a new biological pathway driving IBD and similar conditions that could be targeted with MEK inhibitors.
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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.
news
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.
whitepaper
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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Enhanced NPC cultivation furthers understanding of kidney disease
Induced NPCs facilitate the creation of patient-specific organoid models and improve identification of nephron targeted drugs.
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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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Uncovering the functional role of sRNAs in bacterial adaptation
Researchers find how antibiotic treatments influence the microbiota, providing new strategies for preventing antibiotic-induced damage.
podcasts
CRISPR/Cas: exploring its impact on gene editing
Tune in to this episode where we discuss the opportunities CRISPR/Cas technology offers to revolutionise drug discovery.
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Improved gene editing method for mitochondrial genetic diseases
Researchers have developed the world’s first animal model with A-to-G mtDNA edits achieved using engineered TALEDs.