Gold Nanoparticles Deliver CRISPR in Muscular Dystrophy Treatment

Sophia Ktori, GEN News Highlights, 04 October 2017, http://www.genengnews.com/gen-news-highlights/crispr-nanoparticles-repair-duchenne-muscular-dystrophy-gene/81255009

Scientists working on CRISPR delivery have developed a gold nanoparticle that encapsulates the CRISPR/Cas machinery for delivery to cells.  This new technique, coined CRISPR-Gold, was published in Nature Biomedical Engineering (https://www.nature.com/articles/s41551-017-0137-2).  In the paper the authors demonstrated CRISPR-Gold’s ability to correct the mutated dystrophin gene in a mouse model, with mice receiving CRISPR-Gold treatment displaying two-fold improvement in hanging time in a four-limb hanging test, compared to control mice.

CRISPR Delivery in the Ear

Antonio Regalado, MIT Technology Review, 22 September 2017, https://www.technologyreview.com/s/608899/the-easiest-place-to-use-crispr-might-be-in-your-ear/

One of the major challenges with CRISPR based medical therapies is delivering the necessary components.  One group at the Massachusetts Eye and Ear Infirmary are working on delivering the CRISPR/Cas machinery into mice ears to cure a genetic defect that results in gradual hearing loss.  In the best cases the mice retained significant hearing at two months of age.

Chinese Research Team Fix Genetic Disorder in Human Embryos

David Cyranoski, 02 October 2017, Nature News, http://www.nature.com/news/chinese-scientists-fix-genetic-disorder-in-cloned-human-embryos-1.22694

A new report in Protein and Cell (https://www.ncbi.nlm.nih.gov/pubmed/28942539) is the latest in a string of human embryo CRISPR publications.  Using a modified CRISPR/Cas9 system tethered to a second enzyme that can swap individual DNA bases, the researchers targeted an A to G point mutation that results in β-thalassemia.  Eight of the 20 cloned embryos contained a corrected copy of the gene, possibly curing the recessive disorder.  The scientists were careful to point out that not all cells in the embryo were modified, which could have unintended consequences.

Getting CRISPR into the Body

Emily Mullin, MIT Technology Review, 22 September 2017, https://www.technologyreview.com/s/608898/five-ways-to-get-crispr-into-the-body/

While CRISPR has shown great promise in the lab, its clinical application is more difficult due to delivery challenges.  Researchers are diligently working on this problem, with this article covering five possible ways to deliver the CRISPR/Cas machinery including gels/creams, edible CRISPR, ear injections, skin grafts, and ex vivo therapy.

CRISPR Used to Understand Human Embryos’ Days

Heidi Ledford, Nature News, 20 September 2017, http://www.nature.com/news/crispr-used-to-peer-into-human-embryos-first-days-1.22646

Developmental biologists in the United Kingdom have identified differences in the OCT4 gene between mouse and human embryos by using CRISPR/Cas9 to disrupt OCT4.  While disruption of OCT4 results in abnormal embryo development in both mice and human embryos, human embryos lacking the protein quit developing earlier than mouse embryos and showed a completely different pattern of gene expression.  Additionally, the OCT4 human embryo mutant showed abnormalities in cells that give rise to the placenta.  The researchers hope their work can be sued to improve IVF treatments and explain why some pregnancies fail.

It’s Time for Your Nobel Prize Predictions

Sharon Begley, STAT, 25 September 2017, https://www.statnews.com/2017/09/25/nobel-prize-predictions/

The season of Nobel Prize awards has arrived, and with it comes a slew of predictions.  This year, STAT has identified who they believe has the best chance of winning the Nobel Prize in Medicine; including the CRISPR crowd of Emmanuelle Charpentier, George Church, Jennifer Doudna, and Feng Zhang.  The only problem being each Nobel Prize can only be awarded to three people.

CRISPR Reveals How Butterflies Get Their Color

Rachael Lallensack, Nature News, 18 September 2017, http://www.nature.com/news/crispr-reveals-genetic-master-switches-behind-butterfly-wing-patterns-1.22628

Two new studies in the Proceedings of the National Academy of Sciences (http://www.pnas.org/content/early/2017/08/29/1709058114, http://www.pnas.org/content/early/2017/08/29/1708149114) provide insight into butterfly wing color.  The studies identified two genes, WntA is responsible for creation of the coloring pattern and borders, while optix fills the color within the borders.  Understanding butterfly coloration could provide insights into adaptations such as mimicry.

CRISPR Used to Alter Flower Color

David Nield, Science Alert, 9 September 2017, https://www.sciencealert.com/now-scientists-are-using-crispr-to-change-the-colour-of-flowers

The Japanese morning glory plant has traditionally had violet flowers, however using CRISPR to disrupt a single gene, scientists have altered the flower color to white.  White morning glories can be found; however, it took 850 years for the white version to appear. CRISPR accomplished the task in less than 12 months.  This is the first time CRISPR has been used to alter flower color in higher plants.

Researchers Crowd Source CRISPR On/Off Switch

Stanford Medicine News Center, 29 August 2017, http://med.stanford.edu/news/all-news/2017/08/online-game-challenges-players-to-design-on-off-switch-for-crispr.html

Researchers at Stanford University School of Medicine have created a new online computer game called Eterna where players design RNA molecules that could act as an on/off switch for Cas9.  Molecular biologists at Stanford will then create the most promising molecules and test them in living cells. Researchers aim to have 100,000 players contribute 10 solutions each.  As the research team tests the molecules in the lab, they will provide information to the players for further refinement.

Ethical and Regulatory CRISPR Quagmire in the US

Julia Franz and Katie Hiler, WUNC Science Friday, 27 August 2017, http://wunc.org/post/new-developments-human-gene-editing-face-ethical-and-regulatory-quagmire-us#stream/0

Despite the results of August’s CRISPR edited embryo paper being called into question, its publication has resulted in an increase in the ethics debate.  Scientists agree that CRISPR gene editing will continue to improve and society must grapple with the ethical problems.  Ira Flatow sits down with the author of the August Nature article and with Kelly Ormond, genetics professor at Stanford University and member of the Stanford Center for Biomedical Ethics, to discuss the results and how to proceed.