Your missing links are here (20 November 2020)

Some of the best stuff we picked up around the internet

By: Karen O'Hanlon Cohrt - Nov. 20, 2020

Top Picks

How are heart researchers using the Nobel Prize-winning genetic scissors? UK-focused piece from the British Heart Foundation.
Scientists at Tel Aviv University develop lipid nanoparticle-based CRISPR delivery system that specifically targets and destroys metastatic cancer cells in living animals. The team observed increased survival in aggressive mouse glioblastoma and ovarian cancer models following a single CRISPR treatment. This is “the first example of targeted CRISPR-Cas9 therapeutic gene editing for treating metastatic tumors,” write the authors in the article that was published in Science Advances this week.

Research

Retrons, mysterious complexes of DNA, RNA, and protein found in some bacteria have puzzled scientists for many years. A paper published in Cell earlier this month claims that retrons, just like CRISPR systems, are part of the bacterial immune system to protect bacteria against phage. The new findings are discussed in Science Magazine.
International team of scientists uses CRISPR to engineer primary mouse B cells to produce protective antibodies against HIV. The cells are engineered ex vivo, but when re-injected back into mice, they multiply upon vaccination with HIV-derived epitopes, leading to prolonged protective immune responses and memory B cell production.
Researchers in Germany and Sweden use catalytically dead Cas9 (dCas9) to aid the conversion of glial cells, which are abundant in brain tissue, into new neurons, by activating the expression of neuron-rich mitochondrial proteins during the conversion process. This could be a huge breakthrough in the development of regenerative medicine therapies for acquired brain injury. The findings were published this week in Cell Stem Cell.
CRISPR-Cas interrogation of most of the genes in the human genome reveal important clues about the cellular mechanisms used to degrade microRNAs. MicroRNAs are small regulatory RNA molecules and the findings, published in Science this week, represent an important advance in basic science that may eventually open new doors within medicine.

Industry & Clinical

Harvard University spinout 64x Bio Emerges from Stealth to Drive an Evolution in Cell and Gene Therapy Manufacturing. Among its biggest assests is its proprietary VectorSelect^TMplatform, which dramatically increases experimental throughput to permit screening of mutant cell lines with superior viral vector production capabilities for use in cell and gene therapy applications.
California-based CRISPR biotech Caribou Biosciences announces licensing agreement with Memorial Sloan Kettering Cancer Center (New York) for the fully human anti-CD371 scFvs antibody to enable development of allogeneic cell therapies. The agreement sees Caribou gaining the rights to anti-CD371 scFvs and associated intellectual property within the field of allogeneic CD371-targeted cell therapies, and expands Caribou’s pipeline of allogeneic cell therapies for blood cancers.
Beam Therapeutics present first preclinical data highlighting base editing program for glycogen storage disease Type Ia (GSD1a) at American Association for the Study of Liver Diseases (AASLD) virtual meeting last week. There are currently no approved treatments for this disease, and Beam’s novel adenine base editors have achieved high in vitro and in vivo correction rates for the two most prevalent mutations in GSD1a in preclinical studies.
Locus Biosciences in North Carolina has just bagged almost $1 billion in investment deals. The company has already set a few records since its inception in 2018, going from petri dish to the first ever clinical trials for a highly innovative CRISPR-Cas3 enhanced bacteriophage therapy to combat multiresistant bacteria. We interviewed Joseph Dixon of Locus Biosciences about the trial earlier this year.
FDA lifts clinical hold on MELANI-01 study evaluating Cellectis’ CRISPR-engineered CAR-T therapy UCARTCS1 in multiple myeloma. This follows a period of cooperation between Cellectis and the FDA, which resulted in adjustments to the MELANI-01 clinical protocol designed to enhance patient safety.

COVID19

Feluda paper strip test kit for COVID-19 set to be made available in 8 metropolitan cities in India. The Feluda kit was developed in India by the Tata group in collaboration with the Apollo group of hospitals. It is rapid, delivering results in less than 1 hour and functions similarly to a home pregnancy test.

Review

‘INDEL detection, the ‘Achilles heel’ of precise genome editing: a survey of methods for accurate profiling of gene editing induced indels’: This new review published in Nucleic Acids Research should simplify the task of identifying one or several methods that will suit the particular need to properly detect and characterise genome editing-induced indels.
Report commentary in National Geographic: How to feed the world without destroying the planet. This piece mentions how CRISPR may contribute to solving the problem of feeding the world's population in a sustainable way.

Pioneers

Prime-editing pioneer Andrew Anzalone is named STAT 2020 Wunderkind. We previously interviewed Andrew about his groundbreaking work on prime editing that made Nature’s Top 10 in 2019.
Other pioneers on this year’s STAT Wunderkind list include Cas13 pioneer Cameron Myhrvold, whose work on COVID-19 surveillance we highlighted earlier this year, and Jennifer Hamilton from Nobel Laureate Jennifer Doudna’s lab at UC Berkeley.
Podcast w. Dr. Geoff von Maltzahn, founder of early-stage life science company Tessera Therapeutics: ‘Gene Writing: New Tech to Correct Disease’. Learn more about Gene Writing^TM, a completely novel approach to gene editing that writes therapeutic messages into the genome to treat diseases at the source.