Lifeboat Foundation

Gene editing approaches promise to treat a range of diseases, but delivering editing agents to cells in animal models and humans safely and efficiently has proven challenging. Now, researchers led by a team at the Broad Institute of MIT and Harvard have developed a way to get gene editing proteins inside cells in animal models with high enough efficiency to show therapeutic benefit.

In new work published in Cell, the team shows how they have engineered virus-like particles to deliver base editors — proteins that make programmable single-letter changes in DNA — and CRISPR-Cas9 nuclease, a protein that cuts DNA at targeted sites in the genome. In collaboration with ​​research teams led by Krzysztof Palczewski at the University of California, Irvine, and Kiran Musunuru at the Perelman School of Medicine at the University of Pennsylvania, the team used their particles, called engineered virus-like particles (eVLPs), to disable a gene in mice that can be associated with high cholesterol levels, and partially restored visual function to mice harboring a mutation that causes genetic blindness.

Researchers have developed virus-like particles that deliver therapeutic levels of protein to animal models of disease.

Continue reading “Engineered particles efficiently deliver gene editing proteins to cells in mice” »

In a world medical first, a 57-year-old patient with terminal heart disease received a successful transplant of a genetically-modified pig heart and is still doing well four days later.

A study published by researchers at the University of Illinois Chicago describes a new method for analyzing pyroptosis–the process of cell death that is usually caused by infections and results in excess inflammation in the body–and shows that process, long thought to be irreversible once initiated, can in fact be halted and controlled.

The discovery, which is reported in Nature Communications, means that scientists have a new way to study diseases that are related to malfunctioning cell death processes, like some cancers, and infections that can be complicated by out-of-control inflammation caused by the process. These infections include sepsis, for example, and acute respiratory distress syndrome, which is among the major complications of COVID-19 illness.

Pyroptosis is a series of biochemical reactions that uses gasdermin, a protein, to open large pores in the cell membrane and destabilize the cell. To understand more about this process, the UIC researchers designed an “optogenetic” gasdermin by genetically engineering the protein to respond to light.

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When scientists discovered DNA

DNA, or deoxyribonucleic acid, is a molecule composed of two long strands of nucleotides that coil around each other to form a double helix. It is the hereditary material in humans and almost all other organisms that carries genetic instructions for development, functioning, growth, and reproduction. Nearly every cell in a person’s body has the same DNA. Most DNA is located in the cell nucleus (where it is called nuclear DNA), but a small amount of DNA can also be found in the mitochondria (where it is called mitochondrial DNA or mtDNA).

Houston, TX — Oct 8, 2020 - In a letter published today in the New England Journal of Medicine, a team of physicians from Baylor College of Medicine, Texas Children’s Hospital, and the University of California, San Francisco, describe a remarkable case of a Type 1 diabetes (T1D) patient, who no longer needs insulin to maintain optimal blood sugar levels. The physicians employed a precision/personalized medicine approach to specifically target the underlying genetic mutation, which was the primary driver of this patient’s diabetes.

“To the best of our knowledge, this is the first example of a T1D patient who has experienced a complete reversal of insulin-dependence and we are excited by the prospect that that could be a viable therapeutic strategy for a subset of T1D patients” said corresponding author Dr. Lisa R. Forbes, deputy director for clinical services and community outreach for the Texas Children’s William T. Shearer Center for Human Immunobiology and assistant professor of Pediatrics, Immunology, Allergy and Retrovirology at Baylor.

T1D is a chronic condition in which the pancreas produces little to no insulin, a hormone that maintains sugar levels in the blood. Currently, the treatment options available to T1D patients consist of managing blood sugar levels with insulin, diet and exercise to prevent further complications.

A man with terminal heart disease is responding well three days after being given a genetically modified pig heart in a first-of-its-kind surgery, his doctors reported on Monday.
The surgery, performed by a team at the University of Maryland Medicine in the United States, is among the first to demonstrate the feasibility of a pig-to-human heart transplant, a field made possible by new gene editing tools.
If proven successful, scientists hope pig organs could help alleviate shortages of donor organs.
For David Bennett, a 57-year-old from Maryland, the heart transplant was his last option.

Al Jazeera’s Barbara Angopa reports.

Continue reading “US man recovering after ‘breakthrough’ pig heart transplant” »

Researchers filtered the air around two zoos and identified genetic material from dozens of species, a technique that could help track and conserve wildlife.

I am happy to say that my recently published computational COVID-19 research has been featured in a major news article by HPCwire! I led this research as CTO of Conduit. My team utilized one of the world’s top supercomputers (Frontera) to study the mechanisms by which the coronavirus’s M proteins and E proteins facilitate budding, an understudied part of the SARS-CoV-2 life cycle. Our results may provide the foundation for new ways of designing antiviral treatments which interfere with budding. Thank you to Ryan Robinson (Conduit’s CEO) and my computational team: Ankush Singhal, Shafat M., David Hill, Jr., Tamer Elkholy, Kayode Ezike, and Ricky Williams.

Conduit, created by MIT graduate (and current CEO) Ryan Robinson, was founded in 2017. But it might not have been until a few years later, when the pandemic started, that Conduit may have found its true calling. While Conduit €™s commercial division is busy developing a Covid-19 test called nanoSPLASH, its nonprofit arm was granted access to one of the most powerful supercomputers in the world €”Frontera, at the Texas Advanced Computing Center (TACC) €”to model the €œbudding € process of SARS-CoV-2.

Budding, the researchers explained, is how the virus €™ genetic material is encapsulated in a spherical envelope €”and the process is key to the virus €™ ability to infect. Despite that, they say, it has hitherto been poorly understood:

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It depends.

Warp drive. Site-to-site transporter technology. A vast network of interstellar wormholes that take us to bountiful alien worlds. Beyond a hefty holiday wish-list, the ideas presented to us in sci-fi franchises like Gene Roddenberry’s “Star Trek” have inspired countless millions to dream of a time when humans have used technology to rise above the everyday limits of nature, and explore the universe.

Continue reading “Exotic Forces: Do Tractor Beams Break the Laws of Physics?” »