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Intellia and its research collaborator, IRCCS Ospedale San Raffaele presented new in vitro data showing that CRISPR/Cas9 editing resulted in over 98% knockout of the endogenous T cell receptor (TCR), while achieving transfer of various Wilms’ Tumor 1 (WT1)-specific TCRs into over 95% of isolated T cells.

Intellia Therapeutics is a leading genome editing company focused on developing curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients’ diseased cells.

SOURCES- Intellia Written By Brian Wang. Nextbigfuture.com

Continue reading “Intellia Fixed 95-98% T-Cells Inside the Body Using CRISPR Gene Therapy” »

Here, doctors extract a patient’s own T cells, a type of white blood cell that normally acts as the body’s watcher against cancer and infection. Cancer cells eventually learn to evade T cells or disarm the troops—while turning their own surrounding normal cells into cancerous ones, thus expanding their tumor legion.

CAR-T uses gene therapy to recharge those beaten-down T cells. The UPenn study, for example, relies on a neutered HIV-like virus to deliver an artificial “tracker” protein into those cells. These designer trackers expertly hunt down a protein dubbed NY-ESO-1, which dot certain cancer cells’ surface like a homing beacon.

CRISPR amplifies the CAR-T effect: the team is using the gene editing tool to erase three different “brakes” in T cells. Killing off the first two, TCR α and TCR β, keeps the edited cells in check to prevent friendly autoimmune fire, and allows the added “tracker proteins” to thrive in large numbers. Wiping out the third, PD-1, prevents a phenomenon called T cell exhaustion. It’s aptly named: here, tumor cells secrete molecules that literally shut down T cell activity, zapping away their killing power.

Continue reading “CRISPR Used in Human Trials for the First Time in the US” »

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” »

Scientists from Trinity College Dublin have discovered a potential new target for regulating inflammation, which drives a range of diseases including diabetes, cancer and Alzheimer’s. The potential target is an ancient immune protein—SARM—that has been conserved throughout evolution and thus is very similar in humans, other mammals, flies and worms.

The scientists, from Trinity’s School of Biochemistry and Immunology based at the Trinity Biomedical Sciences Institute (TBSI), discovered a previously unknown but important role that SARM plays in the immune response. Their work has been published today in the prestigious journal Immunity.

In Jules Verne’s famous classic 20,000 Leagues Under the Sea, the iconic submarine Nautilus disappears into the Moskenstraumen, a massive whirlpool off the coast of Norway. In space, stars spiral around black holes; on Earth, swirling cyclones, tornadoes and dust devils rip across the land.

All these phenomena have a vortex shape, which is commonly found in nature, from galaxies to milk stirred into coffee. In the subatomic world, a stream of elementary particles or energy will spiral around a fixed axis like the tip of a corkscrew. When particles move like this, they form what we call “vortex beams.” These beams imply that the particle has a well-defined orbital angular momentum, which describes the rotation of a particle around a fixed point.

Thus, vortex beams can give us new ways of interacting with matter, e.g. enhanced sensitivity to magnetic fields in sensors, or generating new absorption channels for the interaction between radiation and tissue in medical treatments (e.g. radiotherapy). But vortex beams also enable new channels in basic interactions among elementary particles, promising new insights into the inner structure of particles such as neutrons, protons or ions.

Continue reading “Twisting whirlpools of electrons” »

About 1.5 million people died of tuberculosis (TB) in 2017, making it the most lethal infectious disease worldwide. A growing rise in drug-resistant TB is a major obstacle to successfully treating the illness.

Now, researchers at Washington University School of Medicine in St. Louis and Umea University in Sweden have found a compound that prevents and even reverses resistance to isoniazid, the most widely used antibiotic for treating tuberculosis.

The research, published the week of May 6 in Proceedings of the National Academy of Sciences, was conducted in bacteria growing in the lab, setting the stage for future studies in animals and people.

Continue reading “Drug-resistant tuberculosis reversed in lab” »

A discovery opens the possibility of one day restoring loss of vision by activating the retina’s ability to regenerate. Researchers at Baylor College of Medicine, the Cardiovascular Research Institute and the Texas Heart Institute reveal in the journal Cell Reports that although the mammalian retina—a layer of specialized nerve cells that mediates vision and is located on the back of the eye- does not spontaneously regenerate, it has a regenerative capacity that is kept dormant by a cellular mechanism called the Hippo pathway. The discovery opens the possibility of activating the retina’s ability to restore lost vision by manipulating this pathway.

“Damage to the retina can lead to irreparable loss of vision in humans and other mammals because their retinas do not regenerate,” said lead author Dr. Ross A. Poché, assistant professor of molecular physiology and biophysics and member of the Dan L Duncan Comprehensive Cancer Center at Baylor. “However, other animals such as zebrafish can reverse blindness thanks to specialized cells in the retina called Müller glial cells. When the retina is damaged, Müller glial cells proliferate and differentiate into the lost retinal neurons, effectively replacing injured cells with fully functional ones.”

Although Müller glial cells in injured mammalian retina do not restore vision as their counterpart in zebrafish do, other researchers have shown that, when the mammalian retina is injured, a small subset of Müller glial cells takes the first steps needed to enter the proliferation cycle, such as acquiring molecular markers scientists expect to see in a proliferating cell.

Continue reading “Researchers uncover mechanism blocking retina regeneration” »

Research into an intricate toxin delivery system found in bacteria could overcome the problem of pesticide resistance in insects, and might even lead to new cancer treatments.

An international team led by Dr. Michael Landsberg at The University of Queensland has revealed the detailed inner workings of the newest member of a family of naturally occurring insecticidal toxins.

“This toxin, known as YenTc, is a highly effective toxin-delivering nanomachine,” Dr. Landsberg said.

Continue reading “Bacterial toxin research could improve pesticides and help treat cancer” »

Airborne lead from recycled car batteries at the Exide plant in Vernon ended up in the baby teeth of children living nearby, a USC study shows.

“We found the higher the level of lead in the soil, the higher the amount of lead in baby teeth,” said first author Jill Johnston, an assistant professor of preventive medicine at the Keck School of Medicine of USC. “There’s no safe level of lead; it’s a potent neurotoxin. Our study provides insight into the legacy of the impact of industrial contamination on children.”

The Exide plant, located just southeast of downtown Los Angeles, recycled 11 million auto batteries per year and released 3,500 tons of lead until it closed in March 2015 as part of a legal settlement for hazardous waste violations.

Continue reading “Lead contamination found in baby teeth of children living near Exide battery plant” »