image: Dedicated to the science and applications of gene editing.
Credit: Mary Ann Liebert Inc., publishers
The CRISPR Journal announces the publication of its February 2024 issue. The CRISPR Journal is devoted to publishing outstanding research in CRISPR biology, technology, and genome editing. Chief Editor is Professor Rodolphe Barrangou, PhD (North Carolina State University); Executive Editor is Dr. Kevin Davies. For full-text copies of articles or to arrange interviews with the editors, authors, or members of the editorial board, contact Kathryn Ryan at the Publisher.
1. Warrior spirit: An interview with sickle cell pioneer Victoria Gray,
The gene editing world and the sickle cell disease (SCD) community of about 100,000 patients in the US have been celebrating the landmark approval in December 2023 of Casgevy, the CRISPR-based cell therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics. The first SCD patient to enroll in the exa-cel trial in the US was Victoria Gray, from Forest, Mississippi. She received her gene-edited stem cells in July 2019 and has enjoyed a remarkable recovery, free from pain crises, blood transfusions, or hospitalizations. Although she has shared some details of her journey on National Public Radio, she has not given a major interview to the media – until now.
During a virtual summit broadcast from last month, GEN's The State of Cell and Gene Therapy, Victoria spoke candidly with Kevin Davies (The CRISPR Journal) and Uduak Thomas (GEN) about her experiences with SCD, her decision to enroll in the clinical trial, the highs and lows of the lengthy and arduous procedure, and her life after therapy. The CRISPR Journal is publishing a very lightly edited version of that inspiring interview.
Ms. Gray is also featured on the cover of the February 2024 issue of The CRISPR Journal.
Contact: Kevin Davies, PhD (Executive Editor, The CRISPR Journal)
2 Prime editing stem cell lines for type 2 diabetes research
A long-standing research interest of Dr. Francis Collins, former director of the NIH and chief science advisor to the White House), is understanding the genetic basis of type 2 diabetes (T2D). In the February 2024 issue of The CRISPR Journal, Collins, Lori Bonnycastle, and colleagues at the National Human Genome Research Institute use prime editing to generate human isogenic iPSC (induced pluripotent stem cell lines) that provide key models for studying T2D.
Representing some two years of research optimizing the prime editing of single base changes in iPSCs, the Collins lab has largely overcome some long-standing technical challenges. The authors report efficiencies of up to 73 percent for installing different single-base edits in half-a-dozen T2D candidate genes. The streamlined workflow generates edited cell lines within five weeks. Bonnycastle, Collins, and colleagues believe their experience will greatly help other researchers overcome efficiency problems that have hampered research.
Contact: Sarah Bates, PhD (Communications Director, NHGRI)
3. Purging Pigs of PRRS (Porcine Reproductive and Respiratory Syndrome) virus
A team of scientists at Genus, a British animal genetics company, report the generation of pigs resistant to porcine reproductive and respiratory syndrome (PRRS) virus for breeding and food production. Also known as blue-ear pig disease, PRRS was first detected in the US in 1987. It has since spread around the world, with a devastating impact on the swine industry, with estimates of annual losses exceeding $600 million in the US alone.
Using CRISPR-Cas9 genome editing, scientists at Genus edited the genome of pig zygotes and through further breeding of mosaic pigs, successfully established a breeding population of gene-edited pigs with a small deletion in CD163, the cell-surface portal for the PRRS virus. The Genus team considers this to be an important milestone, marking potentially the first integration of CRISPR gene editing into a livestock breeding program that paves the way for the elimination of a major infectious disease in the swine industry.
Contact: Brian Burger (Genus plc)