Your missing links are here 26 June 2020

Top picks

• UK Scientists CRISPR-edited human embryos in the lab, and the experiment went horribly wrong. The study posted on a preprint server bioRxiv on June 5 raises major safety concerns for gene-edited babies.
• Nature news adds more: »CRISPR gene editing in human embryos wreaks chromosomal mayhem«.
• A year in, 1st patient to get gene editing for Sickle Cell Disease is thriving. And here is our story on the new data from the landmark CRISPR-Cas9 gene-editing clinical trial.

Research

• CHANGE-seq, a high-throughput and straightforward approach to understanding the specificity of genome editors.
• Guide-free Cas9 from pathogenic Campylobacter jejuni bacteria plays an active role in attacking human cells rather than in viral defence.
• CRISPR-Cas9 Gene Editing may correct genetic defect in DEB, study suggests.
• Nature-Inspired CRISPR Enzyme discoveries vastly expand genome editing.
• Human stem cells and CRISPR are used to identify degeneration targets that may lead to novel therapeutic approaches for glaucoma.
• Muscle finding could lead to new therapies for heart disease and skeletal muscle disorders.
• Method to combat Parkinson’s disease by astrocyte-to-neuron conversion.
• Multitasking for base editors.

Reviews

• Nature Biotechnology Review from the Liu Lab on the new CRISPR-Cas genome editing tools like base- and prime-editors, and key considerations for choosing genome editing agents.

COVID19

• UC Riverside engineers are using supercomputers to investigate rapid, CRISPR-based COVID-19 tests.
• CRISPR-Cas9 screen reveals host genes that aid SARS-CoV-2 infection. The discovery could assist in the development of new therapies, and reveal why some people are more susceptible to COVID-19 than others.
• How medicine makers personal and professional lives have changed over the course of the COVID-19 crisis. Featuring Simon Tyler at CatSci, Nick Shackley at Johnson Matthey, Chris Lowe at Horizon Discovery, and Andrew Bulpin at Merck.

Clinical trials

• Editas Medicine presents pre-clinical data from a study of EDIT-301 with Sickle Patient Cells for the potential treatment of Sickle Cell Disease.
• CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001™ in Severe Hemoglobinopathies at the 25th Annual European Hematology Association (EHA) Congress.

Vision and opinion

• World Economic Forum 17 bids on how technology could change the world by 2025, including from Rahul Dhanda, Co-Founder and CEO of Sherlock Biosciences. »Engineering biology, machine learning and the sharing economy will establish a framework for decentralising the healthcare continuum, moving it from institutions to the individual.« We recently interviewed Dhanda about the first-ever FDA-approved CRISPR kit for COVID19 detection.

• The Conversation: Gene therapy and CRISPR strategies for curing blindness (Yes, you read that right).

Industry

• CRISPR Therapeutics is joining the rush to build new cell therapy manufacturing space.
• From academia to start-up. Stefano Stella talks about taking groundbreaking CRISPR research to investors for the Cas12a start-up Twelve Bio he co-founded. Stella recently gave us an interview on Cas12a and Twelve Bio here.
• Verve banks $63M to move gene-editing heart attack treatment toward the clinic.
• Exosome delivery tech in $72.5M neuromuscular disease deal.

Heh, huh, wow

• Meet the family working with scientists to fight rare diseases with CRISPR therapy. Video [04:43 min].