Your missing links are here (11 June 2020)

Top Picks

• First gene-edited CRISPR COVID-19 mice. Opens door to study infections and develop therapies.
• This little CRISPR went to market. Gene-editing technology is heading towards commercial reality in pharmaceuticals, food and organ transplant.
• Fantastic and incredible story on a quest to cure a genetic disease so rare, just one person in the world is known to have it. One bad gene: Harnessing genomic pioneering to save Caroline Fletcher.
• Sherlock Biosciences and IDT collaborate on large scale production of Sherlocks first-ever FDA-approved CRISPR kit for COVID19 detection. We recently wrote about the kit here.
• Regeneron and Intellia expand deal to develop haemophilia A and B treatments using CRISPR-Cas9.

Clinical Trials

• New CRISPR clinical trial posted. A clinical trial on advanced hepatocellular carcinoma from Central South University in China using PD-1 knockout engineered T cells.
• Gene-edited T cells to treat diabetes inch closer to clinical trials. Researchers edit the genes of patients' T cells to turn off the destructive autoimmune response in Type 1 diabetes. Another approach using CRISPR-Cas9 was recently described.

COVID19

• PacMan versus COVID19 - scientists are collaborating on a way to deliver CRISPR gene targeting system to patient cells and shred the virus.
• Rapid home-based CRISPR COVID19 tests are coming together in research labs.
• CSIC researchers will use CRISPR tools to destroy the COVID-19 coronavirus genome.
• Big gene therapy names line up behind experimental Covid-19 vaccine.
• In these COVID times with work- and home-life lines becoming blurred, here are some tips on how to cope with that 'always-on' feeling.

Industry

• Impactful linking of two innovative technologies: Single-cell transcriptomics and pooled CRISPR screening. MilliporeSigma and 10x Genomics collaborate on new CRISPR screen on single cells to identify novel therapeutic targets and accelerate drug discovery.
• Avectas and UC Davis collaborate on cell engineering technology. Using Avectas' non-viral Solupure delivery platform, they will study multiple cargo types, including DNA editing cargo.
• Interview with genome-editing entrepreneur Alexis Rovner, co-founder of 64-x, on George Church giving her confidence to follow her dreams and tips for leading a startup.

Research

• Very fast CRISPR on demand. Spatial and temporal control of Cas9 using light-activation. The new study lets researchers study the cellular response to double-strand breaks in real-time.
• CRISPR base editing let deaf mice hear again. The technique could pave the way for human treatment.
• CRISPR-Cas9 of murine cell surface proteins screen reveals a receptor (CXCR4) that is essential for AML (acute myeloid leukaemia) growth and survival.
• CRISPR researchers develop a dual-deaminase CRISPR base editor that enables concurrent adenine and cytosine editing.
• Development of CRISPR-Cas13a-based antimicrobials capable of sequence-specific killing of target bacteria.
• Researchers use in vivo CRISPR screening to discover key gene essential for the survival of leukaemia stem cells.
• Good news for AAV-based gene therapy. AAV-antibodies are a central challenge of AAV gene therapy, and now researchers provide a potential solution. The results, published in Nature Medicine, could allow for those with pre-existing antibodies to pursue gene therapy as well as enable the ability to re-administer AAV vectors.

Safety

• Unique cancer tumour mutations may signal safe CRISPR deployment.
• Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards.
• Johns Hopkins researchers unveil a new paradigm for the intrinsic regulation of CRISPR-Cas systems. In a bioRxiv paper, they show how a long-form tracrRNA is widely used to self-repress the CRISPR-Cas system and serve to prevent auto-immunity.
• Suppression of unwanted CRISPR-Cas9 editing by co-administration of catalytically inactivating truncated guide RNAs.

Vision and opinion

• BIO 2020: Cell and gene therapies will dominate medicine in 30 years.

Heh, huh, wow

• AI and CRISPR - the future is here! Israeli CRISPR-IL consortium to develop advanced tools for better efficiency and accuracy in genome editing of plants, animals and humans. Among the wide variety of companies is TargetGene, whose gene-editing platform TGEE we wrote about here.