CRISPR Therapeutics

A CRISPR-based therapy for the metabolic liver disease phenylketonuria (PKU) has moved one step forward. In vivo prime editing in neonate mice...

Most pharmaceuticals don't reach the brain, leaving cognitive impairments due to diseases like mucopolysaccharidoses (MPS) hard to treat. Now, a...

In a CRISPR first, scientists in Canada have used bacterial conjugation to deliver a CRISPR system to antibiotic-resistant bacteria in the mouse gut....

Chinese researchers have invented a novel system, AdBlue, to construct adenovirus vectors for CRISPR-Cas9 mediated gene editing. The novel system is...

Two weeks ago, we hosted a CMN webinar on CRISPR Delivery Systems. Several hundred attendees from all continents tuned in for the live event. Here's a...

Using microneedles for transdermal delivery of CRIPSR-Cas9 and glucocorticoids researchers have found a viable way to precisely treat inflammation...

Nicole Paulk is at the forefront of adeno-associated virus technology, often used for delivering CRISPR therapeutics. Here Paulk gives CRISPR Medicine...

Erik Sontheimer, Professor and Vice Chair at the RNA Therapeutics Institute and at the University of Massachusetts Medical School talks to CRISPR...

Pharmaceutical and biological research as well as biologics manufacturing rely on genetically modified cell lines with genes that have been modified...

A novel, highly potent lipid nanoparticle (LNP) CRISPR-Cas9 delivery system targets the liver more efficiently than previous methods. Used in...

The mechanism our immune system uses to fend off viruses also attacks AAV vectors. Adding telomeric sequences to these vectors helps them to fly under...

Andre Watson, the founder of the gene delivery platform company Ligandal, quickly pivoted his company to develop a therapeutic for COVID-19. But he...

Lack of efficient delivery vehicles has so far hampered CRISPR-Cas9 editing in non-liver tumours. With a new lipid nanoparticle delivery system,...

California-based gene-editing company Scribe Therapeutics recently unveiled its plans to develop the most advanced platform for breakthrough...

All gene therapies have their own advantages and limitations, but one shared feature is the challenge in delivering the gene-editing or replacement...

For CRISPR genome editing, it is important to consider what form the agent is delivered. In this explainer, we look at the different forms (plasmid...

Using 'dead' dCas9 fused to transcriptional activators researchers wake silent genes to alleviate blindness in a mouse model of retinitis pigmentosa -...

Researchers at UC Berkeley have discovered CasΦ, a small and highly compact CRISPR system in bacteriophages. The new CasΦ works in human cells and...

The first clinical trial results indicate that CRISPR is a safe and beneficial gene editing tool in humans. However, full therapeutic translation of...

CRISPR holds the key to novel genetic therapies but safe and efficient delivery of CRISPR reagents remains a major challenge. US-based researchers...

Continuous advances in gene editing technologies provide hopes for better therapies and cures for genetic diseases. However, a few obstacles remain,...

Advances in cellular and gene therapies are set to revolutionise modern medicine. However, translating the underlying technologies into broadly...

By combining peptide display and different AAV serotypes, researchers have learned how to make and identify viral vectors that can target and...

In a world's first, Locus Biosciences has enrolled the first patients in a clinical trial using bacteriophage boosted with CRISPR to precision-kill...

Gene therapy stops deadly chemical warfare nerve agents in mice. The new research could lead to long-lasting protection by arming mice with...

Safe and efficient delivery tools are crucial for succesful gene therapy. Adeno-associated virus vectors (AAV's) has so far been accepted by the FDA...

Safety director Roberto Nitsch, AstraZeneca, discusses the key safety aspects of delivering potential CRISPR-medicine to patients.