The goal of gene therapy is to permanently cure hereditary diseases. One of the most promising technologies for this is the ...
Cas9 is directed to its target within the DNA sequence through the use of the guide RNA. A specific sequence of DNA that is between two and five nucleotides in length must align with the 3’ end of the ...
CRISPR, the gene-editing technology that has revolutionized biological research, is finally available as a medical treatment with regulatory approval. On December 8 the U.S. Food and Drug ...
For a decade, leading academic institutes and their associated companies fought a bruising, headline-grabbing fight over who held patent rights to CRISPR-Cas9, the revolutionary genome editing tool.
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 ...