Your missing links are here (4 September 2020)
By: Karen O'Hanlon Cohrt - Sep. 4, 2020
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Top Picks
- Two years after the CRISPR baby scandal, an international expert panel reports that it’s still too soon to attempt to make genetically modified babies. The recommendation is not based on ethics but on science. Read Nature’s coverage of the report here.
- In spite of the above, Russian scientist aims to correct hearing loss mutation in unborn babies. Let’s hope we’re not heading for another CRISPR babies scandal.
- Researchers in the USA develop new CRISPR tool that can suppress immune responses to viral delivery vectors used in gene therapy. SafeGen Therapeutics, a spinout from the research team aims to take the new CRISPR tool to the clinic for safer gene therapy.
Research
- New DNA sequencing method, targeted individual DNA molecule sequencing (IDMseq), can accurately detect a single mutation in a pool of 10,000 cells. As more clinical-stage CRISPR studies emerge such a tool will allow high-resolution off-target analysis to detect rare mutations caused by CRISPR.
- CRISPR-Cas13 used to knockdown selected mRNAs in zebrafish embryos, two other fish species and mice. Both embryonic and maternal transcripts (present in the embryo during early embryonic development) could be suppressed, making this technique a relevant tool for studying gene function during early embryonic development.
- Scientists have developed a tool called fluorescent in vivo editing reporter or FIVER, which provides a visual readout of genome editing events by making cells fluoresce in different colours depending on what kind of edits have occurred. It works in primary cells, organoids and in vivo. Work shared on preprint server BioRxiv.
- Chinese researchers develop a CRISPR-Cas13a biosensor with potential in early-stage diagnostics via miRNA biomarker detection. The biosensor can detect and discriminate miRNAs with high sensitivity from cell lysates and serum.
Industry
- New Nevada-based biotech Fixx Pharmaceuticals Inc. launches this week with plans to develop CRISPR and other gene editing therapies for bleeding disorders such as haemophilia. The company hopes to begin its first clinical trial in Q4 of this year using customised zinc finger nucleases to restore factor VIII and IX in haemophilia patients.
- CRISPR Therapeutics (CRSP) is dominating the gene-editing field and is highly attractive to investors. But it is a bit hyped? Here, journalist Dana Blankenhorn looks at the company’s status and advises investors to spread the risk when investing in early stage companies: ‘CRSP is a reasonable speculation. Just don’t put money into it you can’t afford to lose. You may get a gusher, pardner, or a stream of tobacco juice on your wingtips.’
COVID
- The SARS-CoV-2 pandemic has catapulted CRISPR’s role in diagnostics. Here’s a great roundup on the current state of affairs in COVID-19 testing which describes the noteworthy CRISPR-Cas approaches. The first CRISPR application to gain FDA approval ever is a Cas13-based SARS-CoV-2 testing platform called SHERLOCK. You can catch our interview with Rahul Dhanda, CEO of Sherlock Biosciences here.
Safety
- Six leading CRISPR researchers discuss the potentials and limitations of CRISPR gene editing, data robustness, reproducibility, safety (off-target), delivery and more.
Reviews
- CRISPR Journal: Translating CRISPR-Cas Therapeutics: Approaches and Challenges.
Pioneers
- Scientist Luke Gilbert Wins Wachtel Award for Unbiased CRISPR Mapping of Complex Cancer Gene Networks.
- In this Science Rehashed podcast, renowned American Geneticist Dr. George Church shares his opinions on CRISPR editing of human embryos, his project to revive the extinct woolly mammoth, and his journey as a scientific pioneer.
Conferences and Webinars
- Free upcoming webinar – September 15th with industrial presenters covering what it will take to make CRISPR reagents suitable for use in human therapies, as these technologies progress from bench to clinic. Details and sign up here.
- International CRISPR and Gene Editing Symposium is a complementary half-day virtual event running on September 23rd that brings together prominent gene editing researchers to discuss the current state and future of gene editing. Keynote speakers: Alexis Komor, PhD (UCSD) and Matthew Porteus, MD, PhD (Stanford). Details and registration here.
Wow
- Have you heard about Human Nature, a documentary exploring the science, history, and ethics of CRISPR technology? On September 9th, American science series NOVA will premiere a 90-minute special of the documentary. And on September 16th, CRISPR pioneer Jennifer Doudna and Human Nature Director Adam Bolt will chat to MIT Technology Review about the current state of CRISPR and how the documentary was brought to life. Find details here.
- Chile poised to tackle food shortages and climate change with ‘Golden Apple’ and other CRISPR-edited crops.
Tags
CLINICAL TRIALS
IND Enabling
Phase I
Phase II
Phase III
Gastric Cancer and Colorectal Cancer, CRC, (NCT07166263)
Sponsors:
Base Therapeutics (Shanghai) Co., Ltd.
Sponsors:
Base Therapeutics (Shanghai) Co., Ltd.
IND Enabling
Phase I
Phase II
Phase III
Relapsed or Refractory Acute Myeloid Leukemia, AML, (NCT06541444)
Sponsors:
Base Therapeutics (Shanghai) Co., Ltd.
Sponsors:
Base Therapeutics (Shanghai) Co., Ltd.
IND Enabling
Phase I
Phase II
Phase III







