Human trials for CRISPR based sickle cell treatment planned

Julie Steenhuysen, Reuters, http://www.reuters.com/article/us-gene-editing-crispr-idUSKBN13300A

Scientists from Stanford University have been able to reverse sickle cell formation using stem cells derived from patients.  With this proof of concept in hand, Dr. Matthew Porteus, senior author of the study, plans to approach the FDA with pre-clinical data demonstrating that this method is ready for clinical trials.  The plan is to use CRISPR technology to edit stem cells outside of the patient and then reintroduce them.  When this procedure was performed in mice enough of modified cells thrived in the bone marrow to potentially cure sickle cell disease.

Author: Advanced Analytical

Advanced Analytical Technologies, Inc. (AATI) simplifies complex genomics workflows to accelerate research and discovery in pharmaceuticals, life science, biofuels, biotechnology and healthcare.

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