Your Missing Links Are Here (26 March 2021)
By: Karen O'Hanlon Cohrt - Mar. 26, 2021
- Scientists in the US and Germany report persistent repression of tau expression in the brain using engineered zinc finger protein transcription factors. Tau suppression rescued neuronal damage around amyloid plaques in a mouse model of Alzheimer’s disease. The findings were published in Science Advances and are the results of Sangamo Therapeutics’ preclinical tauopathies programme in partnership with Biogen.
- Researchers use CRISPR to edit haematopoietic cells to generate a disease model for acute erythroid leukaemia (AEL), a rare form of acute myeloid leukaemia (AML) with insuffienct preclinical models and poor prognosis. The new model recapitulated human AEL, shed light on disease mechanisms, and was used for drug screening with known anti-cancer drugs. The findings were published yesterday in Blood.
- A team at Purdue University, US, has uncovered the structural basis for substrate recognition and cleavage by the dimerisation-dependent CRISPR-Cas12f nuclease, which is also known as Cas14. Cas12f is much smaller than other Type V Cas endonucleases and the newest results may facilitate genome editing applications using this miniature Cas. The findings were published yesterday in Nucleic Acids Research.
- Intellia Therapeutics presents the first preclinical data on its novel cytosine deaminase base editor technology at the 7th Cold Spring Harbor Laboratory virtual scientific meeting on Nucleic Acid Therapies this week. The data demonstrates that Intellia’s base editor technology in combination with its proprietary cell engineering process achieved >90% T cell editing efficiency (multiple knockouts simultaneously) while maintaining translocations at background levels.
- Bioheng Biotech, a Chinese clinical-stage biotech company, raises $80 million in Series B financing to advance its allogeneic immuno-cell therapy pipeline. The company is developing CRISPR-edited CAR T-cell therapies for the treatment of a number of blood and solid cancers.
- BioCentriq, the New Jersey Innovation Institute’s cell and gene therapy development and manufacturing center, announces a new partnership with Kytopen, a Cambridge-based MIT spinout. The partnership will combine Kytopen’s proprietary Flowfect® technology, which uses electro-mechanical energy for non-viral payload delivery, with BioCentriq’s cell and gene therapy manufacturing capabilities to develop new curative autologous and allogeneic therapies.
- Researchers in Singapore engineer a CRISPR-Cas12a variant that enables robust and rapid testing of wild-type and mutant SARS-CoV-2 strains without the need for RNA purification. Using hybrid DNA-RNA guides greatly increased the reaction rate, bringing the test completion time to below 30 minutes. The findings were recently published in Nature Communications.
- CRISPR-based detection of SARS-CoV-2: A review from sample to result. This review from researchers at Pennsylvania State University, US, summarises the CRISPR-based diagnostic approaches that have emerged during the COVID-19 pandemic. Future perspectives of CRISPR-based sensing towards point-of-care molecular diagnosis applications are also addressed.
- Active Delivery of CRISPR System Using Targetable or Controllable Nanocarriers. This review summarises and discusses the targetable and controllable nanocarriers that are being used to actively deliver CRISPR cargo to cells.
- CRISPR Medicine News will soon host its first webinar: Certainty after CRISPR will discuss the expected and unexpected genetic modifications that may occur during and after CRISPR-Cas9 editing, as well as how one can detect and validate these. Date: April 20, 2021. More details and signup here.
- Gene inactivation by CRISPRi: Modulate gene expression with PAM-anchored targeting. A sponsored webinar by Horizon Discovery on March 31, 2021. Find webinar registration details here.
- Aarhus University in Denmark are hosting a virtual Bachelor's level course (5 ECTS) in genetic engineering using CRISPR-Cas from 9-20th August. The course introducues state-of-the-art CRISPR-Cas systems and key applications in genetic engineering with different practical online workshops. Application deadline is 8 April. See here for more information and signup details.