Some of the best stuff we picked up around the internet
By: Gorm Palmgren - Feb. 19, 2021
Top picks
Bluebird bio has halted a clinical trial after two patients developed leukaemia-like cancer. The trial used gene therapy to treat sickle cell disease by introducing a functional gene - and thus, it is not a kind of gene editing. The cancer is not necessarily linked to the gene therapy, but might have been caused by the chemotherapy treatment used to prepare patients for the ex vivo treated blood stem cells.
An international research team led by Rory Johnson from University College Dublin, Ireland, has significantly enhanced the efficiency of CRISPR-Cas9 deletion. The enhancement is obtained by repressing the DNA-dependent protein kinase catalytic subunit (DNA-PKcs) that controls an early step in nonhomologous end-joining (NHEJ). The new method works across diverse cell lines, gene delivery methods, commercial inhibitors, and guide RNAs.
Other Chinese researchers have demonstrated a method that uses paired Cas9 nickases to generate iPSC lines with different gene dosages of duplicated genes. The technique enabled the generation of iPSC lines with monoallelic, biallelic, or triallelic knock-out of the amyloid precursor protein (APP) gene in Alzheimer's patients carrying three copies of the gene. The approach opens up for the development of new clinical therapeutics for dementia.
A promising method to inhibit the progression of lung adenocarcinoma in vivo has been shown in xenografts. The results were achieved by CRISPR interference (CRISPRi) targeting the novel, noncoding oncogenic driver FAM83H‐AS1.
David Liu and colleagues have developed new software, PrimeDesign, for the rapid and simplified design of prime editing guide RNAs. The authors used the software to construct a comprehensive and searchable database that includes candidate prime editing guide RNA (pegRNA) and nicking sgRNA (ngRNA) combinations for installing or correcting >68,500 pathogenic human genetic variants.
Excision BioTherapeutics announces the completion of a $60 million financing to advance CRISPR-based HIV-treatment. As a result of the funding, the Company's lead candidate EBT-101, which targets and deactivates viral genes, will go into a phase I/II clinical trial in patients with chronic HIV infection.
As expected, CRISPR Therapeutics has announced substantial losses of $107.0 million for the fourth quarter of 2020 and $348.9 million for the year ended December 31, 2020. The decrease is primarily attributable to the sale of certain licenses under the Company's collaboration with Vertex Pharmaceuticals during 2019.
Cytovia Therapeutics and Cellectis have announced a strategic research and development collaboration to develop TALEN gene-edited induced pluripotent stem cells (iPSCs) natural killer (NK) and chimeric antigen receptor (CAR)-NK cells. The partnership's financial terms include up to $760 million of development, regulatory, and sales milestones from Cytovia to Cellectis for the first 5 TALEN gene-edited iPSC-derived NK products.
CRISPR stocks have soared 4-5 times in the last few years, so the question now is: should you buy or should you sell? Forbes gives you some pieces of advice.
An opinion piece in Scientific American addresses the consequences of using CRISPR and other gene-editing techniques to "reduce human diversity and increase social inequality by editing out the kinds of people that [...] are understood as having bad genes."The piece is written by Sandy Sufian, associate professor of health humanities and history at UIC School of Medicine, and Rosemarie Garland-Thomson, professor of English at Emory University, who were both born with severe genetic conditions.
Carl June from the University of Pennsylvania, US, is among this year's Dan David Prize laureates that were announced this week. June is a pioneer in CRISPR-based cancer therapies, and CRISPR Medicine News interviewed him last year. The seven laureates share $3 million in the three categories: History of Health and Medicine, Public Health, and Molecular Medicine.
A webinar arranged by Illumina and 10x Genomics will address the advances in single-cell CRISPR. Researchers Shiqi 'Russell' Xie from Genentech and Lea Starita from the University of Washington will discuss the matter on 26 February.
Research work to produce Ghana's first gene-edited crop with increased vitamin A content has begun at the University of Cape Coast. The project will use CRISPR to knock out the production of an enzyme that converts beta carotene - a precursor of vitamin A - into other products. Sweet potato is very resilient, has a high yield and is adaptable to many environmental conditions expected from climate change.