Your Missing Links Are Here (26 February 2021)

Top picks

• Decoding the CRISPR-baby stories. Piece from MIT Technology Review about three books that explore the He Jiankui scandal and what gene editing means for the future of humanity.
• The Jin-Soo Kim lab at Seoul National University demonstrates in embryo base editing of mitochondrial DNA in mice using DddA-TALE fusions. The researchers used base editing to create mitochondrial disease models in mice and the work highlights the potential of base editing to treat mitochondrial disorders. The findings were published in Nature this week.

Research

• Scientists from Leipzig University, Germany use DNA origami, in which special DNA sequences are used to form complex three-dimensional nanostructures instead of a simple double helix, to measure the activity of CRISPR enzymes on an ultra-fast timescale.
• Researchers at Dresden University of Technology, Germany, have developed Cre-Controlled CRISPR, a new method for CRISPR that makes it possible to turn off a single target gene in one specific cell type of choice. The new method combines the best parts of the popular Cre/lox system with CRISPR-Cas9 and the findings were recently published in Nature Communications.
• Researchers at Rutgers University, US, have developed Pin-point^TM, a new base-editing system that deploys RNA aptamers within the gRNA to position DNA-modifying enzymes at genomic targets. Pin-point^TM can perform point-mutation edits with high precision and with low on-target INDEL formation. The findings were published in the CRISPR Journal this week.
• Research led at the Wellcome Sanger Institute, UK, has uncovered almost 143,000 species of bacteriophage living in healthy human guts. The results of this work, which also form the basis for the newly established Gut Phage Database were recently published in Cell.
• Researchers in Germany describe CAST-Seq (single targeted linker-mediated PCR sequencing), a new preclinical assay to sensitively identify and quantify chromosomal aberrations derived from on-target and off-target activities of CRISPR-Cas nucleases or TALENS, respectively, in human hematopoietic stem cells. The results were published in Cell Stem Cell this week.
• Researchers in China and the USA develop nanobody-based CAR T-cells using CRISPR-Cas9 technology for solid tumour immunotherapy. The CAR T-cells were developed using CRISPR-Cas9 technology to deliver the anti-CD105 coding seqeuence via an adeno-associated virus and the resulting anti-CD105 CAR T-cell cells reduced tumour burden and improved survival in tumour-bearing mice and demonstrated anti-tumour activity in human tumour xenograft models. CD105 is emerging as an important sold tumour target, and the findings of this study were published in Nature.

Industry

• Precision Biosciences, US, announced 3-year preclinical study results demonstrating long-term durability and safety of in vivo gene editing with their meganuclease-based platform ARCUS to reduce LDL cholesterol levels in nonhuman primates. The study results were published in Molecular Therapy.
• Beam Therapeutics announces acquisition of Guide Therapeutics. The takeover of Guide Therapeutics’ advanced lipid nanoparticle (LNP) and proprietary in vivo LNP screening technology will allow for further expansion of Beam Therapeutics’ genetic medicines into new target tissues and diseases.
• Editas Medicine announced fourth quarter and full year 2020 results and updates on ongoing programmes yesterday. You can catch the update here.
• Intellia Therapeutics also announced fourth quarter and full year results from 2020 yesterday. You can find that update here.
• Last further quarter update for this week comes from Allogene Therapeutics, who also released its full year results from 2020 yesterday with updates on their programmes for gene-edited CAR T therapies for cancer. Read Allogene’s update here.

Clinical trials

• Promising clinical trial data from Locus Biosciences, Inc. on the world's first clinical trial for a CRISPR-enhanced bacteriophage therapy. Read our interview with Locus Bioscience’s Chief Scientific Officer Dave Ousterout, PhD on the new results and the future of CRISPR as a new antibiotic.

COVID-19

• Researchers in Germany and Switzerland publish genome-wide CRISPR screens in human cells lines infected with either a highly pathogenic or an endemic coronavirus. Genome-wide loss-of-function mutations revealed host defence factors that are required for the replication of both endemic and emerging coronaviruses. Three of these bind to certain immunosuppressive agents and were found to be inhibited by already approved drugs in vitro in the study. The findings, which were published on the preprint server BioXriv this week, highlight a number of host factors that may be targetable by already approved drugs to treat coronavirus infections.
• A FIND webinar series on COVID-19 Diagnostics. Webinar on 5th March 2021 covering insights on Feluda – the first indigenously CRISPR-based SARS-CoV-2 test, developed in India. FIND is the Foundation for Innovative New Diagnostics, a global NGO based in Switzerland. Register for the webinar here.

Conferences and events

• Precision Engineering of the Genome, Epigenome and Transcriptome. Virtual Keystone Symposia March 8-10, 2021. See event programme here and register for the event here.

Meetings and webinars

• The CRISPR Revolution: The Power & Promise of Gene Editing. Nobel Laureate Jennifer Doudna to feature at inaugural Lemley Family Leadership Lecture Series at Brown University next month. Register for the online event here.
• Mapping the Worldwide Research, Innovation, and Diffusion Activity of CRISPR. Webinar hosted by University of Oregon as part of Knight Campus Entrepreneurship Speaker Series. The speaker, Dr. Samantha Zyontz, is a Fellow at the Center for Law and the Biosciences and a Research Fellow of Intellectual Property at Stanford Law School. Register for the webinar here.
• CRISPR-Cas9 genome editing: gene therapy and beyond. Online workshop hosted by GENoMiX on 6th March 2021. Register for the event here. Note that this is not a free event.

Reviews and roundups

• The race for CRISPR-based therapeutics. Roundup of the three leading gene-editing companies in the CRISPR Medicine space; CRISPR Therapeutics, Intellia Therapeutics, and Editas Medicine.
• After the Nobel, what next for CRISPR gene-editing therapies? An easy read that brings you up to date on the status of clinical developments within the CRISPR therapy field, highlights the potential and some of the key challenges that must be solved before the use of CRISPR Medicine can become widespread.
• Book Review of ‘CRISPR People: The Science and Ethics of Editing Humans’ (MIT Press, 2021) by Henry T. Greely. Review by Michael A. Goldman of San Francisco State University for Science Mag.

Opinion

• Hotter, drier, CRISPR: editing for climate change. Scientists at The University of Queensland believe that gene editing technology will play a vital role in climate-proofing future crops to protect global food supplies.