Your Missing Links Are Here (12 February 2021)

Top picks

• Cas13-based CRISPR test for SARS-CoV-2 from UC Santa Barbara shows high sensitivity for asymptomatic screening. The test, known as CREST, combines the specificity of CRISPR and the sensitivity and robustness of PCR with easy-to-access cheap reagents and equipment, and the most recent findings were published this week. We interviewed the lead researcher to learn about the development CREST last year.
• Researchers from the US, UK and Brazil have created tiny, brain-like ‘organoids’ that contain a gene variant harboured by two extinct human relatives, Neanderthals and Denisovans. The researchers used CRISPR–Cas9 to introduce the extinct form of a gene called NOVA1 into human pluripotent stem cells and cultured these further to develop brain organoids. It is hoped that these Neanderthal-like mini brains can shed light on the genetic pathways that allowed human brains to evolve. The findings were published in Science today.

Research

• Research led at the Icahn School of Medicine at Mount Sinai has utilised CRISPR to build a cellular model that recapitulates early to late stage evolution of acute myeloid leukemia. The researchers used CRISPR to make step-wise driver mutations that lead to malignancy, and then mapped transcriptional and chromatic changes over time, revealing certain events that may be worth targeting therapeutically. The findings were published in Cell Stem Cell earlier this week.
• Scientists from ChristianaCare’s Gene Editing Institute (US) have developed an affordable, downloadable app that scans for potential off-target edits that may occur when CRISPR is used to correct disease-causing mutations. The open-source app, known as DECODR, is based on an algorithm that can predict off-target edits through analysis of sequence data. The findings were published in The CRISPR Journal this week, and a free version of the software is available here.
• Researchers from Rice University in the US have built a programmable chromatin kinase by directly fusing nuclease-dead Cas9 (dCas9) to a hyperactive, truncated variant of the human MSK1 histone kinase. The programmable kinase, called dCas9-dMSK1, can target specific histones and influence gene activation. The work represents a step forward in epigenome editing and the findings were published in Nature Communications this week.
• The Kleinstiver Lab at Massachusetts General Hospital and Harvard Medical School have published their protocol for the HT-PAMDA assay, a high-throughput PAM determination that can characterise hundreds of Cas enzymes in parallel.

Industry

• AbbVie and Caribou Biosciences have announced a collaboration and license agreement for two next-generation off-the-shelf CAR-T cell products. The $40M deal will see Caribou’s next-generation Cas12a CRISPR hybrid RNA-DNA (chRDNA) genome editing and cell therapy technologies used to engineer CAR-T cells that can withstand host immune attack. AbbVie will lead all clinical development, commercialisation, and manufacturing efforts. You can read the companies’ press release here.
• Boston-based biotech Ensoma launched this week with $70M Series A funding and a partnership with Takeda Pharmaceuticals (Japan). The partners will develop a suite of off-the-shelf genomic medicines, using Ensoma’s new Engenious vectors to deliver multiple types of genetic cargo to patients, including gene-editing treatments, gene therapies and gene regulators.
• Ex-Biogen chief and Editas Medicine chair James Mullen becomes new CEO of Editas Medicine. He replaces current CEO Cynthia Collins from the 15th February 2021.

Vision, opinion and interviews

• Stat interviews James Mullen, Editas's new CEO about why he took the job and his vision for the future.
• ABC News speaks with a doctor and a patient Victoria Gray, who are involved in Vertex Pharmaceuticals' and CRISPR Therapeutics' clinical trial for CRISPR therapy CTX001 to treat sickle cell disease.

Pioneers

• The 6th International Day of Women and Girls in Science day was held yesterday. To mark the day, here is a roundup of 22 female scientists who whose historical scientific contributions have changed our world forever.
• University of Californa, Los Angeles is to establish a new center for precision genetic medicine, thanks to a $29M gift from real estate developer and philanthropist Allen Ginsburg and his wife Charlotte. The Allen and Charlotte Ginsburg Center for Precision Genomic Medicine will host researchers and clinicians to study the role of genetics in disease and develop new therapies for genetic disorders.

COVID-19

• Researchers at NSF International and Novateur Ventures screen more than than 1,100 independently assessed, point-of-care COVID-19 tests. From this, they identified 5 direct (antigen/RNA) tests for detection of acute infection and 6 indirect (antibody) tests for detection of prior infection that meet the WHO’s "desirable" Target Product Profile (TPP) criteria for COVID-19 diagnosis. Mammoth Biosciences’ SARS-CoV-2 Detectr Test was the 2^nd best direct test.

Review

• CRISPR/Cas technology as a promising weapon to combat viral infections. This review discusses recent advances and future prospects in the use of CRISPR to treat and diagnose viral deleterious infections such as HIV or SARS‐CoV‐2.
• Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy. This review discusses the potential of using cutting-edge genetic approaches to treat Duchenne muscular distrophy and addresses outstanding challenges that must be overcome before new genetic therapies become clinically possible.
• Applications of CRISPR genome editing to advance the next generation of adoptive cell therapies for cancer. A detailed review describing the CRISPR-based genetic modifications that can improve the efficacy, cost-effectiveness and scalability of adoptive cell therapies for cancer.

Heh, huh wow

• Get healthy drinking CRISPRed beer? Scientists from the Biological Center of the Czech Academy of Sciences in České Budějovice are using CRISPR to modify certain genes to boost the production of prenylated flavonoids, a group of natural compounds that have anti-cancer, anti-inflammatory and anti-bacterial properties. Although the idea of therapeutic beer is fascinating, current EU biotech restrictions are blocking further development.