What matters in CRISPR medicine!

It's been a great start to the decade for CRISPR genome editing and gene therapy, and we got You covered on CRISPR Medicine News.

Here is what we have been writing.

The first CRISPR gene therapy is safe
In November, researchers announced the results of the first US CRISPR gene therapy on three patients. Now the data are out in a peer-reviewed paper. Here is our story with comments from leading researcher Carl June and experts in the field.

In vivo genome and base editing with no detectable off-target mutations
Marcello Maresca, the very friendly genome editing wiz at AstraZeneca, gave us an interview. He talks about doing in vivo CRISPR genome editing without detectable off-target mutations and using CRISPR to make disease models and gene therapy.

Anti-CRISPR proteins: A smart way to make gene therapy safer
The CRISPR kill-switch can make gene therapy safer. The latest research describes a new principle of kinetic insulation using anti-CRISPR proteins to increase the specificity of CRISPR-Cas genome editing by tenfold or more.

Gene therapy against lethal chemical warfare nerve agents
Last but not least, 'roundup' soldiers may be a thing in the future. US Army researchers develop an innovative gene therapy in mice that could give soldiers long-lasting protection against lethal nerve agents such as sarin.

More good reads

Top picks

• RNA editing is taking off. CRISPR make room.
• Interview with Carl June on CRISPR, CAR–T and how the Vietnam War dropped him into medicine.

• Using a CRISPR–Cas9 genome-wide screen to uncover unique T-cell, that gives hope for universal cancer therapies.

• Crispr'd Cells Show Promise in First US Human Safety Trial. Using genetically-edited cells to supercharge the immune system caused no adverse effects in cancer patients. It's too soon to tell if it can be a cure.

• Parents entering baby Eva in a controversial lottery to win $2.1 Mio. gene therapy treatment.

• Pricey gene therapies fuel debate over drug prices - and why there is no easy fix.

• Paying for CRISPR Cures: The Economics of Genetic Therapies.

Research

• Building better base editors, new research from the Liu-lab brings base-editors that make fewer off-targets and access more genome locations.

• A new CRISPR-based tool can probe and control several genetic circuits at once.

• A CRISPR cure for cystic fibrosis?

• Unwanted CRISPR-Cas9 duplications and a warning to researchers.

• Opening hidden DNA to CRISPR. Custom-built molecules enable the editing of genes previously obscured by closed chromatin.

• Discovery could lead to the first treatment for the dry form of age-related macular degeneration (AMD) and improve treatment for the wet form.

• Simple strategy increases CRISPR knock-in efficiency by chemically modifying DNA-ends.

• Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy.

• Researchers identify a novel cellular entry factor for AAV vector types.

Industry

• The European Patent Office (EPO) has affirmed a patent issued to CRISPR pioneer Emmanuelle Charpentier, rejecting arguments filed in opposition by the Broad Institute of MIT and Harvard.

• Base Editing Drug Developer, Raises $180M in IPO.

• Global CRISPR Genome Editing Market Set For Rapid Growth.

• Good news: Gene therapy trial to treat hereditary immune disorder show promising results in six patients.

• Pfizer aims for Phase III Gene Therapy Trials in 2020.

• From bench to bedside - Synthego adopts GMP standards for producing sgRNA.

• Companies Join Forces to Address Urgently Needed Quality Control of CRISPR Genome-editing.

Uh ah wow

• Curious about the new online resource - The Global Gene Regulation Tracker - from the Genetic Literacy Project? Listen to The Talking Biotech podcast.

• UK Parliamentary Office publishes brief on human germline genome editing.

• Biohacking CRISPR, reports on gene editing 'kitchen experiments', EU regulators are struggling to safeguard the new developments.

And that's it

Thank You for now - more great reads are on the way.

And if you have questions or suggestions, you can directly reach us at editor@crisprmedicinenews.com. We love hearing from readers!

(c; Rasmus

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