For some diseases, gene therapies offer the potential for lifelong disease amelioration and even cure. And these immensely important novel biotechnologies may be on the cusp of a boom. That is in part ...
Northwestern scientists have developed a new nanostructure that supercharges CRISPR’s ability to safely and efficiently enter cells, potentially unlocking its full power to treat genetic diseases. By ...
Philadelphia, October 28, 2024 – CRISPR/Cas9 is a gene editing tool that has revolutionized biomedical research and led to the first FDA-approved CRISPR-based gene therapy. However, until now, the ...
Ever since Vertex and CRISPR therapeutics scored a groundbreaking U.K. authorization for their sickle cell gene therapy Casgevy in November, the looming hurdle to commercialization was securing a ...
Researchers from Helmholtz Munich and the Technical University of Munich (TUM) report that they have developed a new tool (Cas13d-NCS) that allows CRISPR RNA molecules that are located within the cell ...
Dr. Musunuru and Dr. Ahrens-Nicklas holding KJ post infusion Senior Correspondent Gene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments ...
A new type of therapy that 'edits' a gene in patients with a rare heart condition has been shown to be safe and effective, according to research from UCL and the Royal Free Hospital. Results for the ...
CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is an advanced technology developed in 2012 that can be used to edit genes. It can be used to find specific DNA sequences inside ...
Researchers discovered the secret checkpoints that keep CRISPR precise, revealing how the tool shifts from inactive to active without misfiring. The insight could transform gene therapy, making ...
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