Discover how CRISPR genome editing is revolutionizing medicine. Learn the science of Cas9, current clinical trials, and the future of gene editing.

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

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 ...

Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly ...

CRISPR–Cas9 is a revolutionary gene-editing tool, adapted from this bacterial immune system, that has been widely explored for its clinical applications in recent times. While the CRISPR–Cas9 shows ...

The approved CRISPR-Cas9 gene therapy involves isolating hematopoietic stem cells (HSCs) from SCD patients and using CRISPR-Cas9 gene editing to reactivate fetal hemoglobin (HbF) genes that are ...

BUFFALO, NY- November 28, 2023 – A new editorial paper was published in Oncotarget's Volume 14 on November 27, 2023, entitled, “The double-edge sword of CRISPR application for in vivo studies.” In ...

The U.S. Food and Drug Administration approved the first cell-based gene therapies for sickle cell disease, including the ...

In December 2020, the New England Journal of Medicine published a paper titled CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia, marking an exciting new chapter in the treatment of ...

Most approved gene therapies today, including those involving CRISPR-Cas9, work their magic on cells removed from the body, after which the edited cells are returned to the patient. This technique is ...