Some of the best stuff we picked up around the internet
By: Karen O'Hanlon Cohrt - Apr. 23, 2021
Beam Therapeutics published new data describing modifications to the traditional base-editing architecture to develop so called next-generation base editors, which can directly and precisely modify the pathogenic mutation in sickle cell disease (SCD). This advance provides hope for the development of autologous base-edited cell therapies for SCD. The findings were published in CRISPR Journal this week.
Researchers in the US design a new CRISPR strategy ‘Co-opting Regulation Bypass Repair’ (CRBR), which unlike standard CRISPR-Cas - that tends to rely on homology-directed repair proteins that are only present in dividing cells - can be used in dividing and non-dividing cells and tissues. CRBR uses non-homologous end joining to insert a sequence between a mutated gene's promoter and the mutated portion of the gene. This essentially hijacks the promoter to express the newly inserted sequence, which could be a wild-type copy of a disease gene. CRBR has been tested in mice and in human tissue cultures, and proof-of-concept data was published in Molecular Therapy earlier this week.
Vertex and Obsidian Therapeutics form collaboration to leverage Obsidian’s cytoDRiVE® platform technology to discover gene-editing therapies whose activity can be precisely regulated using small molecules with Vertex’s scientific and clinical capabilities in small molecule, cell and genetic therapies to more rapidly bring new gene-editing therapies to the clinic.
Researchers in Germany succeed in correcting mutations in β-haemoglobinopathies using CRISPR-Cas9 and adeno-associated virus (AAV)-delivered donor templates. The strategy was tested in patient-derived haematopoietic stem and progenitor cell (HSPCs) with promising rates of gene correction and a significant increase in haemoglobin levels. The findings were published in CRISPR Journal this week.
Chinese researchers show that bubble hairpin single guide RNAs (BH-sgRNAs), which contain a hairpin structure with a bubble region on the 5' end of the guide sequence, can be efficiently applied to both cytosine and adenine base editors and significantly decrease off-target editing without compromising on-target editing efficiency. Their findings were publishd in mBio.
A team in Australia has developed Generalizable On-target activity ANAlyzer (GOANA), a high-throughput web-based software for determining editing efficiency and cataloguing rare outcomes from next-generation sequencing data. GOANA calculates mutation frequency and outcomes relative to a supplied control sample. The findings were published in CRISPR Journal this week and the tool can be accessed online here.
A digital warm-start chip-based CRISPR (dWS-CRISPR) assay for sensitive quantitative detection of SARS-CoV-2 in clinical samples has been developed by researchers at University of Connecticut, US. The assays demonstrates sensitivity and precision in clinical swab and saliva samples and could also detect SAR-CoV-2 in heat-treated saliva without RNA isolation. This work was published in Biosensors and Bioelectronics.
Researchers in Japan have achieved amplification-free SARS-CoV-2 RNA detection with CRISPR-Cas13. The new digital detection platform called SATORI combines CRISPR-Cas13-based RNA detection and microchamber-array technologies and rapidly detects SARS-CoV-2 RNA with high specificity and sensitivity (~5 fM) that was aided by the simulataneous use of multiple different guide RNAs. The findings were recently published in Communications Biology.