Prenatal editing halts progression of Hurler syndrome in pre-clinical model
US researchers used an AAV9 vector to edit a single base mutation in a prenatal mouse model, halting progression of Hurler syndrome.
List view / Grid view
US researchers used an AAV9 vector to edit a single base mutation in a prenatal mouse model, halting progression of Hurler syndrome.
Every manufacturing process for potential biologics begins with cell line development, whether it’s for clinical trials or a market launch.
10 reasons to choose Bethyl antibodies and reagents from Fortis Life Sciences for your applications.
Learn quick technical tips to optimise your ELISA workflow, including how to: select the right ELISA kit, avoid contamination and normalise data.
Chemical synthesis of guide RNAs for CRISPR-Cas9 gene editing enables accurate and rapid production of CRISPR libraries and screening in an arrayed, one-gene-per-well fashion.
Genome editing technologies have given investigators the power to unlock a variety of new applications and experimental approaches. While gene knockouts have been achievable for several years, successful gene knock-ins have remained elusive due to the low efficiency of homology-directed repair (HDR).
Researchers have created an algorithm that can predict the efficiencies of guide RNAs for CRISPR, potentially making it more precise.
Complete solutions for neurological disease research and discovery - helping you to better understand diseases to improve patient outcomes.
Learn about a practical, high-throughput method for validating the target-specificity of antibodies for the application of western blot.
A wide range of tools to support your immuno-oncology research and help redefine and develop tailored, life-changing immunotherapies to fight cancer.
A CRISPR gene editing technique has been developed to restore dystrophin, which is missing in many Duchenne muscular dystrophy (DMD) patients.
Solutions to aid understanding of cellular and molecular pathways in diabetes and translate these findings into prevention and treatment strategies.
11 May 2021 | By PerkinElmer
Get the Big Picture and listen to Pharma thought leaders how they view CETSA® and its impact on their Drug Discovery.
Gain more insight into immune-tumor interactions and learn how antibody detection techniques like multiplexing is advancing immunotherapies.
Scientists have said that researchers need to be more aware of unintended mutations to human embryos following CRISPR-Cas9 genome editing.