Lifeboat Foundation: Safeguarding Humanity
Toggle light / dark theme

Blog

Category: biotech/medical – Page 3

Scientists from Mass General Brigham and Beth Israel Deaconess Medical Center have developed a novel gene editing tool called STITCHR. Unlike traditional CRISPR, STITCHR inserts entire genes at precise locations, minimizing unintended mutations. This gene editing tool simplifies use and offers potential as a one-time treatment for genetic disorders.

The technology uses retrotransposons, naturally occurring “jumping genes” found in all eukaryotic organisms, which can move and integrate into genomes. Using computational screening, the researchers identified and reprogrammed a specific retrotransposon to work with the nickase enzyme from CRISPR, forming the complete STITCHR system that allows a precise, seamless gene insertion into the genome.

STITCHR offers the potential to replace or supplement entire genes, creating a more universal treatment option for various genetic diseases. The research team is now working to improve its efficiency and move it toward clinical use. Their study, published in Nature, highlights how insights from basic cellular biology can drive innovation in genetic medicine and lead to new therapeutic tools.

Read more | >

While CRISPR-mediated gene editing has led to powerful advances across biology, medicine, and agriculture, challenges persist in optimizing the editing efficiency of enzymes, such as the widely used Cas9 nuclease. This is especially true in therapeutic use cases, where the goal is to attain high rates of editing via a relatively low and transient enzyme dose.

In a new study published in the April 2025 issue of The CRISPR Journal titled, “Hairpin Internal Nuclear Localization Signals in CRISPR-Cas9 Enhance Editing in Primary Human Lymphocytes,” researchers from the Innovative Genomics Institute (IGI) at the University of California (UC), Berkeley, present a strategy to improve editing efficiency in human immune cells for therapeutic applications by leveraging new constructs for nuclear localization signal (NLS) sequences.

“Efficient CRISPR enzyme production is essential for translation. This is one element that allowed the rapid clinical evaluation of Casgevy, the world’s first genome editing drug. Unfortunately, this aspect tends to be overlooked in the basic research performed in academia,” said Ross Wilson, PhD, assistant adjunct professor of molecular and cell biology at UC Berkeley, who led the new study.

Read more | >

An INRS research team has identified a new family of enzymes that can make precise cuts in single-stranded DNA. A few years ago, the introduction of CRISPR technology marked a significant breakthrough in the scientific community. Derived from a component of the bacterial immune system, CRISPR ena

Read more | >

In this Perspective, the authors propose that patients with psoriatic arthritis and an inadequate response to therapy can be classified into two distinct subgroups, characterized by persistent inflammatory and non-inflammatory phenotypes, and discuss potential mechanisms underlying these phenotypes, as well as considerations for treatment strategies and trial design.

Read more | >

Many cells in our body have a single primary cilium, a micrometer-long, hair-like organelle protruding from the cell surface that transmits cellular signals. Cilia are important for regulating cellular processes, but because of their small size and number, it has been difficult for scientists to explore cilia in brain cells with traditional techniques, leaving their organization and function unclear.

In a new series of work, researchers at HHMI’s Janelia Research Campus, the Allen Institute, the University of Texas Southwestern Medical Center, and Harvard Medical School used super high-resolution 3D electron microscopy images of mouse brain tissue generated for creating connectomes to get the best look yet at primary cilia.

Read more | >

Non-personalized content and ads are influenced by things like the content you’re currently viewing and your location (ad serving is based on general location). Personalized content and ads can also include things like video recommendations, a customized YouTube homepage, and tailored ads based on past activity, like the videos you watch and the things you search for on YouTube. We also use cookies and data to tailor the experience to be age-appropriate, if relevant.

Select “More options” to see additional information, including details about managing your privacy settings. You can also visit g.co/privacytools at any time.

Read more | >