Lifeboat Foundation

Fourth patent for CRISPR-Cas9 expected in coming months as patent board works its way through past UC applications.

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.

Continue reading “Cryptic mutation is cautionary tale for crop gene editing” »

#Interesting #opinion This was brought up a decade ago, yet he was never asked to alter his genetics to make it fair for others. Is the current case one of discrimination? The recent work on the Chinese CRISPER babies showed that it augmented their ability. Will CRISPER babies also have in the future to “Change Their Genetics” to compete in sports if deemed unfair advantage?

Powerful gene-editing tools have the potential to heal—or to harm. Now there’s a race to develop the antidote to the next bioweapon.

It turns out the capacity for offspring to benefit from their parents’ experiences doesn’t just happen with fish. Munday tells me about Daphnia, often called water fleas, that are found in freshwater lakes, ponds, and puddles. The tiny crustacean can hatch with either a round head or a pointed head. If it shares the water with predators such as fish or midges or other insects, spikes and spines help lessen the likelihood of being eaten. For many species of juvenile water flea…

Insights into epigenetics and inheritance show that some organisms can adapt to a changing world.

CRISPR’d crops could help us address both hunger and obesity.