Your missing links are here (8 January 2021)

Some of the best stuff we picked up around the internet

By: Gorm Palmgren - Jan. 8, 2021

In the future, CRISPR may be key to help rapidly ageing children. Researchers from David Liu's lab describe in a Nature paper how base editing has been successfully used to double the lifespan in a mouse model of the fatal disease progeria that gives young children the appearance of having aged rapidly. We contacted David Liu right before the paper was published, and you can read our interview with him here.
Here at CRISPR Medicine News, we have selected a few remarkable highlights that researchers worldwide have achieved with the CRISPR genome editing tool. Read our commentatory "2020 was an incredible CRISPR year" here.

A new electrochemical microfluidic biosensor has improved detection of microRNAs (miRNAs) that are increasingly used for diagnostics. The new multiplexed CRISPR-Biosensor X divides the biochip's previous single channel into subsections allowing for the amplification-free and simultaneous quantification of up to eight miRNAs.
Phages are the principal target of the adaptive immune system, namely CRISPR, that bacteria and archaea developed as protection from foreign DNA. Studying the CRISPR-Cas spacers in Pseudomonas aeruginosa, researchers now confirm that the system also targets closely related prokaryotic species and that CRISPR acts as an important constraint to horizontal gene transfer.
Researchers have used CRISPR-Cas9 to quickly validate findings from genome-wide association studies (GWASs) that screen large populations for genetic variants linked to a disease or other phenotype. A GWAS study pointed to a putative enhancer element for the gene PRPF38A involved in bone cell formation. When CRISPR-Cas9 was used to delete this sequence, symptoms of a kind of osteoporosis were alleviated.
A new study sheds light on CRISPR-Cas9 editing dynamics in different conditions, including cell types and delivery methods. Among the results, the researchers find that the kinetics of AAV6-mediated homology-directed repair (HDR) deletions are inferior to non-homologous end-joining (NHEJ).

Intellia Therapeutics has outlined its expected 2021 milestones and strategies. These include clinical validation of a treatment for transthyretin amyloidosis (ATTR) and a lipid nanoparticle delivery system as well as expanding the company's pipeline of new therapy candidates for genetic diseases and cancers.

A new test for the rapid detection of Sars-CoV-2 specific RNA has been developed by Jennifer Doudna's lab and is posted on the medRxiv* server. The test is called DISCoVER (DiagnosticS with Coronavirus Enzymatic Reporting) and uses viral nucleic acid amplification and Cas13-mediated detection without RNA extraction.

A roundup in the New England Journal of Medicine by Jennifer Doudna and others summarises the important advances and exciting possibilities of human molecular genetics and genomics over the last few decades. Starting with Sanger DNA sequencing and PCR over the Human Genome Project and diagnosis of genetic diseases, the journey temporarily ends with CRISPR-Cas9 and related gene editing tools.
A new review elucidates recent advances in the use of CRISPR-Cas technology in airway diseases for correcting genetic defects and developing new therapeutic approaches.
Another review looks at how gRNAs can be engineered to increase stability and guide efficiency, ameliorate inflammatory signalling caused by the CRISPR system, and improve homology-directed repair (HDR) efficiency.
CRISPR-based strategies for the diagnosis and therapy of infectious diseases are the focus for yet another review. The review highlights both the potential benefits and risks of the CRISPR technology.

The Allen Institute is a non-profit institute based in Seattle, and they have made several CRISPR gene-edited fluorescently-tagged cell lines available to the public for research. Among the new additions are hiPSC cell lines that target 37 key cellular structures and substructures.

It is often challenging to efficiently deliver CRISPR reagents to targets cells, especially when targeting the central nervous system due to the blood-brain-barrier (BBB). Researchers have now improved glioblastoma treatment in an in vivo mouse model by locally opening the BBB with ultrasound and subsequently introducing the CRISPR reagents packaged in lipid-polymer hybrid nanoparticles.
A new gene therapy company EnCapsid will focus on developing novel adeno-associated virus (AAV) delivery vectors with improved cargo-capacity and more efficient delivery of CRISPR-Cas9 reagents. EnCapsid is a spin-out of BrickBio and is backed by Tiger Gene LLC.

In 2018, the European Court effectively ruled out CRISPR-edited crops by classifying them as GMOs. However, researchers challenge the ruling's interpretation that defines GMOs as organisms that "have been altered in a way that does not occur naturally". The researchers argue that many edits imposed by CRISPR can occur naturally and therefore crops altered in such ways are not GMOs as defined by the EU.
England, which is now both out of the EU and can set its own rules in agriculture independently of the UK, has launched plans to ease the regulation of CRISPR gene edited crops. The UK that initially opposed the European Court ruling mentioned above has also aired the same consideration.
Researchers have taken the first steps toward uncovering attempts from athletes to use CRISPR-Cas9 for gene doping. They use mass spectrometry to detect fragments of Cas9-protein in blood plasma. So far they have succeeded doing so in plasma spiked with Cas9 in either its active form used for conventional gene editing or in its inactive form used for base or prime editing.