Your Missing Links are here (20 December 2019)

Some of the best stuff we picked up around the Internet

- Dec. 20, 2019
News

By: Rasmus Kragh Jakobsen

Top picks

The hotly anticipated Nature’s 10 - the annual list of ten people who mattered in science this year. Last year the CRISPR rogue He Jiankui was on the list and again this year a Chinese CRISPR researcher makes the list. Hongkui Deng for the first published report of CRISPR Cas9 gene-editing in a person. CCR5 edited immune cells that are impervious to infection by HIV. Russian CRISPR baby scientist Denis Rebrikov is on the 'to watch in 2020' list.

Prime editing selected among Nature top 10 papers of 2019 - CRISPR tool enables precise genome editing by copying RNA into the genome.

CRISPR Babies: MIT's Technology Review reports exclusive excerpts from He Jiankui’s unseen original research manuscript on creating the gene-edited twins Lulu and Nana.

Great feature on the huge challenges facing gene therapy not just technologically but also ethically.

Research

Great review on advances in genome editing through control of DNA repair pathways by leading CRISPR scientist Jacob E. Corn.

CRISPR gene drive could counter rising antibiotic resistance crisis. A CRISPR-based system targets amplified antibiotic-resistant genes - demonstrates 100-fold improved efficiency in targeting antibiotic-resistant bacteria.

CAR-T modified to resist exhaustion and improve activity against solid tumors in mice. Nature paper.

Gene editing cardiac stem cells protect replacement cells from being attacked by the immune system in vitro.

Delivery

Insight from GEN on in vivo CRISPR delivery options - viral vectors, lipid-based nanoparticles, and polymer formulations.

Machine-guided AAV design lay the foundation for engineering superior and highly tailored delivery vectors for future gene therapies.

A new gene therapy strategy, courtesy of Mother Nature - Scientists turn a natural cellular process into a drug-delivery system.

High-tech method for uniquely targeted gene therapy developed.

Safety

Taking CRISPR Safety to the Next Level. The proteome could be a game changer in detection of genome editing outcomes.

Great new study on rapid screening for unintended CRISPR-induced mutations in the immediate vicinity of the CRISPR repair site. Nature Communications Biology paper underscores the importance of better quality control technologies for precision gene therapy applications.

Off-targets rare in CRISPR/Cas9 zebra fish exome study.

Vision and opinion

How Far Are We from (Accurately and Safely) Editing Human Embryos?

CRISPR this, and CRISPR that. Why is CRISPR constantly in the headlines but RNAi, so much farther ahead, relatively unknown?

And this very nice Milestones in antisense RNA research from Nature.

Industry

Thermo Fisher Unveils Viral Vector Manufacturing Expansion.

Astellas Pharma has agreed to acquire Audentes Therapeutics for approximately $3 billion cash, in a deal the companies say is intended to create a top-tier gene therapy developer.

GenEdit and Editas Medicinesigned a deal.

MilliporeSigma Licenses CRISPR Tech to Promega to develop CRISPR-edited cell lines.

Uh ah wow

60 minutes episode: George Church's lab at Harvard Medical School is working to make humans immune to all viruses, eliminate genetic diseases and reverse the aging process.

A phage that resists all forms of the antiviral defense known as CRISPR has an unusual means of survival.

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Missing Links: Your Missing Links are here (20 December 2019)
Missing Links: Your Missing Links are here (20 December 2019)
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Sickle Cell Disease, SCD, (NCT06287099)
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IND Enabling
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Sickle Cell Disease, SCD, (NCT06300723)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
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