CMN Weekly (9 July 2021)

Top picks

• Dutch researchers have developed a method to engineer large-scale chromosomal deletions in human cells by CRISPR-Cas9 efficiently. The technique employs inducing double-strand breaks at two ends of a chromosomal arm and selecting the cells that have lost the intermittent region. A high frequency of successful deletions (up to 30% of selected clones) suggests an oncogenic advantage of these deletions, and the authors discuss the implications for cancer development and therapy.
• Stanford University spinout, Graphite Bio, is preparing for clinical trials of ex vivo gene editing of hematopoietic stem cells using CRISPR–Cas9 and adeno-associated virus serotype 6 to treat people with sickle-cell disease. The company, co-founded by Matthew Porteus and Maria Grazia Roncarolo, will use a strategy to repair the mutations behind sickle cell rather than insert an additional β-globin gene. A piece in BioPharma Dive reviews the various gene-editing strategies for sickle cell disease.

Industry

• Intellia Therapeutics has announced closing an underwritten public offering of 4,758,620 shares of its common stock at the public offering price of $145.00 per share. The move will bring in approximately $690 million.
• The stocks of the gene-editing company Regeneron Pharmaceuticals rallied 11% in June, and The Motley Fool's James Brumley shares his idea why that happened.
• On the contrary, another major player, CRISPR Therapeutics, lost 10% of its value last week. The team at Trefis has a suggestion as to how this happened and whether the stock will rebounce.

Research

• Researchers from Bristol University describe a new method for high-throughput mapping of single DNA cleavage events. The technique, ENDO-Pore, employs nanopore sequencing of rolling circle amplified cleavage events.

COVID-19

• A new CRISPR-based diagnostic tool for point-of-care COVID-19 detection with the naked eye is described by Chinese researchers. The method uses gold nanoparticles and CRISPR/Cas12a-assisted RT-LAMP and can detect four copies/μL of SARS-CoV-2 RNA in less than 40 minutes.

Reviews

• RNA splicing modulation is an upcoming and promising strategy as therapy for specific genetic diseases like cancer, and it can be achieved using either CRISPR or splice-switching oligonucleotides. A paper by Israelian researchers discusses both the challenges and limitations of these technologies.
• The potential for using CRISPR-Cas9 gene editing to manage Alzheimer's disease (AD) and its clinical manifestations is discussed in a review. In addition, the review looks at how CRISPR has allowed the development of empirical AD models, therapeutic lines, and diagnostic approaches for better understanding the nervous system, from in vitro to in vivo models.
• A review in Current Medical Science summarises the past achievements of CRISPR/Cas9 in CAR-T therapy and focuses on the potential CAR-T targets.

Opinion

• MIT Technology Review has selected the most promising "35 Innovators Under 35" in five categories: Inventors, Entrepreneurs, Visionaries, Humanitarians and Pioneers. Among them is Jonathan Gootenberg for his work on expanding gene editing capabilities and Janice Chen for her contribution to use CRISPR to make new diagnostic tests.
• While gene editing and other innovative biotechnological solutions show remarkable results for treating various diseases, society cannot ignore the cost questions. That is the message from Barbara Ryan, founder of Barbara Ryan Advisors and former member of PhamExec.com's editorial advisory board.
• A paper in the journal Social Studies of Science takes a look at 'irresponsible' research. Using He Jiankui's two gene-edited babies as an example, the author argues that it results from various (dis)qualifications and distinctions. Therefore, it is essential to containing such irresponsible research, the veracity of knowledge claims must be confirmed, and institutions and decision-makers are called to act.
• Another paper in Science and Engineering Ethics also takes off with the CRISPR babies in a discussion of "When is it Safe to Edit the Human Germline?". The author argues that existing policy proposals are inadequate in different ways and suggests alternative measures.
• Yet another paper discusses the potentials and pitfalls of CRISPR and addresses how biotechnology can interact with computer technology and create an upgraded human, which will revolutionize the world of work and education.

Videos and Podcasts

• Stanford Online has published a 52-minute video presentation by the university's Monte Winslow entitled "How CRISPR Is Revolutionizing Cancer Research". Drawing insights from his laboratory and examples from current literature, he shows how this technology has revolutionized our ability to study and treat cancer more efficiently and effectively.
• BBC has published a 47-minute long audio podcast entitled "The Hopes And Hurdles Of CRISPR And Gene Editing". The author, Avery Kleinman, talks about CRISPR as a successful medical and agricultural tool and mentions the significant ethical hurdles that the international scientific community will have to face.