Programmable CRISPR tools are turning stem cell biology into something closer to software engineering, shrinking ...
Development of bespoke therapies has been a driving goal in medical research. This year, the announcement of the first patient treated with personalized CRISPR therapy, Baby KJ, highlights this ...
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 ...
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 ...
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 ...
Scientists introduced CRISPR to the world as a gene-editing tool in summer 2012, when landmark papers from two independent groups demonstrated how the system could be wielded to make cuts in DNA. Now, ...
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 ...