CMN Weekly (26 November 2021) - Your Weekly CRISPR Medicine News
Top Pick
- A news feature in Nature Biotechnology entitled The CRISPR Children speculates about the fate of the the first children with germline-edited genomes. They are now growing up in China after their embryos were manipulated in He Jiankui’s lab three years ago. The author asks how their lives might be affected by their edited genomes, and how emerging knowledge on the potentially genotoxic effects of gene editing may help guide the children's physicians about what to look for.
Research
- American researchers have developed an automated design of CRISPR prime editors (PE) for 56,000 human pathogenic variants. The authors also assessed the possibility of developing universal PE constructs and found that common genetic variants impact only a small minority of designed PEs.
Industry
EdiGene has entered a research collaboration with Peking Union Medical College Hospital in Beijing, China. The two entities will develop gene-editing therapies for inherited retinal degeneration using EdiGene's proprietary LEAPER RNA-editing technology.
CRISPR Therapeutics has been granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX110 by the FDA. CTX110 is an allogeneic CAR-T cell therapy targeting CD19+ B-cell malignancies (see here and here).
Intellia Therapeutics will expand its ongoing clinical trial with the in vivo CRISPR/Cas9 gene-editing therapy NTLA-2001 for transthyretin amyloidosis (ATTR). The study initially included only patients with polyneuropathy (ATTRv-PN), but now also patients with cardiomyopathy (ATTR-CM) will be included. Read more about the clinical trial here and here.
Cytovia and Cellectis have announced an expansion of their iNK partnership in China. iNK uses TALEN gene-edited iPSC-derived NK and CAR-NK cells as a therapy for various cancers.
Viral detection
- A novel biosensor for the detection of human papilloma virus-11 (HPV-11) is described by Turkish researchers. The chronoimpedimetric biosensor employs dead Cas9-modified graphene oxide-PAMAM electrodes and can detect HPV-11 between 50 pM and 1000 pM with good linearity, sensitivity and selectivity.
- A protocol for point-of-care detection of nucleic acid samples of infectious agents in less than an hour is described by American researchers. The method employs (reverse transcription-) recombinase polymerase amplification (RPA) of DNA or RNA, Cas12a-mediated target recognition and cleavage, and subsequent magnetic beads-mediated colorimetric readouts. The method is termed M−CDC.
- Chinese rechers have developed a CISPR-LAMP technology employing Cas12a to detect influenza A virus, influenza B virus, respiratory syncytial A virus, respiratory syncytial B virus, and SARS-CoV-2. In addition, a uracil-DNA-glycosylase-reverse transcription-LAMP system was also developed which can effectively remove dUTP-incorporated LAMP amplicons and reduce the risk of aerosol contamination.
Reviews
- A review by Chinese researchers highlights the application of CRISPR-Cas9 technology in treating neurological disorders. This includes Alzheimer's disease, amyotrophic lateral sclerosis and/or frontotemporal dementia, Duchenne muscular dystrophy, Dravet syndrome, epilepsy, Huntington's disease, and Parkinson's disease.
- Circular DNA (circDNA) has many potential uses in therapeutics and diagnostics, and some of them include exploiting circDNAs as donors in CRISPR-Cas systems. This is the topic of a review by American and Chinese researchers.
- Russian researchers take a look at CRISPR-Cas9 and related technologies to treat hepatitis B virus (HBV), which causes severe liver disease. CRISPR has already demonstrated very high antiviral potency. The review discusses the key issues that must be solved to make CRISPR-Cas9 an anti-HBV therapy.
- A review discusses the potential use of CRISPR-Cas13 in understanding virus-host interactions. The review aims to provide a comprehensive summary of the recent advances of Cas13 for targeting viral RNA for either RNA-mediated degradation or CRISPR-Cas13-based diagnostics.
- Dutch researchers review the modulation of CRISPR-Cas9 genome-editing activity by small molecules. The authors look into the mechanisms of action of the small molecules and suggest that direct-acting small molecules for Cas9 are more suitable for precisely regulating Cas9 activity.
Commentary
- A commentary looks into the recent advances in employing CRISPR to excise latent HIV-1 across the broad spectrum of viruses typically found in infected patients.