HGP – MaxCyte, NIH NIAID Study Published in Science Translational Medicine Demonstrates CRISPR-Cas9 Repair of X-linked Chronic Granulomatous Disease (CGD) Gene
Gaithersburg, MD, January 12, 2017 – MaxCyte®, Inc., a developer and supplier of cell engineering products and technologies to biopharmaceutical firms engaged in cell therapy, drug discovery
and development, biomanufacturing, gene editing and immuno-‐oncology, announced today pre-‐clinical results from a collaborative study with investigators at the National
Institutes of Health’s (NIH) National Institute of Allergy and Infectious Diseases (NIAID). Results from the study demonstrated clinically relevant levels of correction in the CYBB gene
mutation in long-‐term engrafted hematopoietic stem cells (HSC) and differentiated neutrophils in cells obtained from individuals with X-‐linked chronic granulomatous disease (X-‐
The study was published in Science Translational Medicinei and entitled “CRISPR-‐Cas9 gene repair of hematopoietic stem cells from patients with X-‐linked chronic granulomatous
disease.” Using MaxCyte’s patented Flow Electroporation™ Technology, researchers demonstrated that transfecting three molecules, a single-‐strand oligonucleotide correction template, along
with messenger RNA encoding for CRISPR-‐Cas9 gene editing complex and selected guide RNA into HSC obtained from individuals with X-‐CGD, resulted in correction of mutation in the CYBB gene. This correction occurred at clinically relevant levels following long-‐term engraftment in preclinical models.
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