CMN Weekly (24 September 2021)
By: Karen O'Hanlon Cohrt - Sep. 24, 2021
- Researchers in Taiwan compared the ability of an adenosine base editor (ABE) with CRISPR-Cas9 editing to rescue parkinsonism phenotypes in induced pluripotent stem cell-derived dopaminergic neurons carrying the LRRK2 p.G2019S mutation, which is the most prevalent genetic cause of Parkinson's disease. The team found that ABE could directly correct the pathogenic mutation, and the findings were published in Stem Cell Research & Therapy this week.
- Verve Therapeutics announces development of proprietary GalNAc-LNP (lipid nanoparticle) delivery technology enabling efficient in vivo liver delivery of base editors. The GalNAc-targeting ligand facilitates delivery of base editors to the liver, and resulted in high efficiency base editing in mouse models of homozygous familial hypercholesterolemia.
- Verve Therapeutics reports new pre-clinical data with its base editor candidate, VERVE-101 that is developed for the treatment of cardiovascular disease. The new data demonstrate robust, durable and precise editing of the PCSK9 gene 36 non-human primates.
- Beam Therapeutics announces updated pre-clinical data highlighting optimised lipid nanoparticle-based delivery approaches for in vivo base editing to the liver and other tissues. The company also reported initial in vivo data showing delivery capabilities to tissues outside of the liver.
- GenEdit announces $26 million in Series A financing from multiple investors at TIDE 2021 conference about oligonucleotide and peptide therapeutics. The funding will be deployed to advance GenEdit’s NanoGalaxyTM platform of non-viral, non-lipid polymer nanoparticles, and to select therapeutic candidates to treat diseases of the nervous system. and provides research update at TIDES 2021
- Scientists in China have developed a simple and rapid CRISPR-based assay to detect Group B Streptococcus (GBS) in pregnant women. Tranmission of GBS from mother to baby is among the leading causes of neonatal illness and death worldwide. The new assay demonstrated high sensitivity, shorter turnaround times and lower instrument demands compared to PCR-based detection methods. The findings were published in Emerging Infectious Diseases this week.
- A team in China and US have performed embryonic base editing in a mouse model of hypertrophic cardiomyopathy (HCM) using a novel adenosine base editor (ABE) platform. The researchers demonstrated correction of the mutation as well as the phenotype in post-natal mice and their offspring. They also show potential for in vivo base editing via by in utero injection of an adeno-associated viral (AAV)-delivered ABE. The findings were recently published in Circulation Research.
- Researchers at University of California, San Diego, have demonstrated correction of a single nucleotide polymorphism (SNP) highly associated with risk of developing asthma in primary human CD4+ T cells. The SNP contributes to increased expression of the ORMDL sphingolipid biosynthesis regulator 3 protein (and a downstream protein under ORMDL3 regulation), which contributes to increased disease susceptibility. The findings were recently published in Allergy.
- Prime editing is a relatively new gene-editing modality that can induce small genetic changes, including insertions, deletions, all 12 possible point mutations, and combinations of these changes, without the need for donor DNA or double-strand breaks, albeit often with poor efficiency. A team in South Korea and Japan report the development of a more efficient prime editor 2 by addition of the Rad51 DNA-binding domain. The findings were published in Nature Communications yesterday.
- In an attempt to address the difficulties in precise in vivo regulation of CRISPR-Cas9 activity, researchers in China have developed sono-controllable and reactive oxygen species (ROS)-sensitive sonosensitizer-integrated metal–organic frameworks (MOFs), denoted as P/M@CasMTH1. These are developed for augmented sonodynamic therapy (SDT) efficacy using CRISPR-Cas9 genome-editing technology, by facilitating the delivery of CRISPR-Cas9 reagents as well as allowing spatiotemporal regulation of genome editing. The findings were published in Advanced Materials earlier this week.
Conferences and meetings
- World CRISPR Day: The 2nd annual World CRISPR Day happens as a virtual even on October 20th. The day is organised by Synthego, and the goal is to share, listen, and learn about the latest advances in CRISPR, with presentations from leading scientists in the gene-editing space, a panel discussion featuring the women of CRISPR and more! See here for more information and sign-up details.
- Labroots Annual CRISPR Virtual Meeting is scheduled for September 29, 2021. The free event with more than 21,000 expected attendees will explore new trends in CRISPR in biology and disease including gene therapy, oncology and COVID-19, as well as ethics considerations on the medical applications of CRISPR. See here for more information and sign-up details.
Interviews and podcasts
- A conversation with Jennifer Doudna, PhD, developer of CRISPR gene-editing technology. Jennifer Doudna speaks to Association of American Medical Colleges News about her thoughts on CRISPR's future, representation in science and how academia can provide opportunties for innovation.
- Researchers in Saudi Arabia have identified a new Cas13 variant, dubbed miniature Cas13 (mCas13), which they used to design, build, and test a SARS-CoV-2 detection module coupling reverse transcription loop-mediated isothermal amplification (RT-LAMP) with the mCas13 system to detect SARS-CoV-2 in synthetic and clinical samples. The findings were published in ACS Synthetic Biology earlier this week.
- Looking for a job in CRISPR? Then look no further than CMN Jobs where we regularly add new positions within CRISPR medicine, gene therapy and cell therapy. Check out the current listings right here.
Huh, Heh, Wow
- Eliminating beef cattle pregnancy loss with CRISPR/Cas9 technology. Researchers at Texas A&M College of Agriculture and Life Sciences are exploring gene-knockout technology to determine the role of glycoproteins in embryo loss in pregnant cows.
Acute Myeloid Leukemia, AML, (NCT05066165)
Transfusion Dependent Beta-Thalassaemia, TDT, (NCT04925206)
EdiGene (GuangZhou) Inc.
EdiGene (GuangZhou) Inc.
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CAR T for CD19+ hematological cancers, (NCT05037669)
University of Pennsylvania
University of Pennsylvania