Lifeboat Foundation

Targeted genome editing tools, such as meganucleases (MGN), zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALENs) and more recently the clustered regularly interspaced short palindromic repeats (CRISPR) have revolutionized most biomedical research fields. Such tools allow to precisely edit the genome of eukaryotic cells by inducing double-stranded DNA (dsDNA) breaks at specific loci. Relying on the cell endogenous repair pathways, dsDNA breaks can then be repaired by non-homologous end-joining (NHEJ) or homology-directed repair (HDR) allowing the removal or insertion of new genetic information at a desired locus.

Among the above-mentioned tools, CRISPR-Cas9 is currently the most simple and versatile method for genome engineering. Indeed, in the two-component system, the bacterial-derived nuclease Cas9 (for CRISPR-associated protein 9) associates with a single-guide RNA (sgRNA) to target a complementary DNA sequence and induce a dsDNA break. Therefore, by the simple modification of the sgRNA sequence, users can specify the genomic locus to be targeted. Consistent with the great promises of CRISPR-Cas9 for genome engineering and gene therapy, considerable efforts have been made in developing efficient tools to deliver the Cas9 and the sgRNA into target cells ex vivo either by transfection of plasmids coding for the nucleases, transduction with viral-derived vectors coding for the nucleases or by direct injection or electroporation of Cas9-sgRNA complexes into cells.

Researchers have designed Nanoblades, a protein-delivery vector based on friend murine leukemia virus (MLV) that allows the transfer of Cas9-sgRNA ribonucleoproteins (RNPs) to cell lines and primary cells in vitro and in vivo. Nanoblades deliver the ribonucleoprotein cargo in a transient and rapid manner without delivering a transgene and can mediate knock-in in cell lines when complexed with a repair template. Nanoblades can also be programmed with modified Cas9 proteins to mediate transient transcriptional activation of targeted genes.

Continue reading “Nanoblades Are Another Delivery Option for Gene Editing into Live Organisms” »

Even in this “age of the genome,” much about genes remains shrouded in mystery. This is especially true for “cryptic mutations”—mutated genes that are hidden, and have unexpected effects on traits that are only revealed when combined with other mutations. Learning from one infamous cryptic mutation in particular, researchers from CSHL share important lessons for breeding or gene editing in crops.

This story starts with the Campbell Soup Company and a field of tomatoes in the mid 20th century. One particular tomato plant had an unexpected beneficial trait: the fruits separated from the vine right where the green cap and stem touch the rest of the fruit. It turned out that this spontaneous natural mutant was ideal for large-scale production.

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As we dive into the brave new world of gene editing, CRISPR technologies are undoubtedly becoming increasingly precise, but alongside enhanced precision is also the necessity for developing ways to inhibit or block the process – an anti-CRISPR molecule, if you will. New work from the Broad Institute and Brigham and Women’s Hospital has presented a study that homes in on small molecules that may have the ability to safely block the CRISPR gene editing process.

Jose Cordeiro is promoting the development of rejuvenation biotechnologies in Spain and the integration of Latin American immigrants into Spain’s aging society to maintain the country’s productivity. He was at the recent Undoing aging conference in Berlin and gave us an interview about his political goals.

At Undoing Aging 2019, jointly organized by SENS Research Foundation and Forever Healthy Foundation, there was a session focused on the ways to make healthy life extension and medical progress a greater part of the global agenda. Among the speakers there was Jose Cordeiro, the vice chair of Humanity Plus, director of The Millennium Project, fellow of the World Academy of Art and Science, and board member of the Lifeboat Foundation.

Continue reading “An Interview with Jose Cordeiro” »

To understand them well, look to the past.

By Michael Le Page

People with incurable melanomas and brain or breast cancers are to get injections of tumour-fighting viruses.

The trial will test the safety of a virus that has been engineered to shrink tumours – an approach that holds promise for a range of cancers, including deadly brain tumours.

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Bioengineers at Boston Children’s Hospital report the first demonstration of a robot able to navigate autonomously inside the body. In an animal model of cardiac valve repair, the team programmed a robotic catheter to find its way along the walls of a beating, blood-filled heart to a leaky valve—without a surgeon’s guidance. They report their work today in Science Robotics.

Surgeons have used robots operated by joysticks for more than a decade, and teams have shown that tiny robots can be steered through the body by external forces such as magnetism. However, senior investigator Pierre Dupont, Ph.D., chief of Pediatric Cardiac Bioengineering at Boston Children’s, says that to his knowledge, this is the first report of the equivalent of a self-driving car navigating to a desired destination inside the body.

Dupont envisions autonomous robots assisting surgeons in complex operations, reducing fatigue and freeing surgeons to focus on the most difficult maneuvers, improving outcomes.

Continue reading “A first in medical robotics: Autonomous navigation inside the body” »