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.
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.
Bio-Printing Complex Human Tissues & Organs — Dr. Anthony Atala, MD — Director, Wake Forest Institute for Regenerative Medicine, Wake Forest School of Medicine, Wake Forest University.
Dr. Anthony Atala, MD, (https://school.wakehealth.edu/Faculty/A/Anthony-Atala) is the G. Link Professor and Director of the Wake Forest Institute for Regenerative Medicine, and the W. Boyce Professor and Chair of Urology.
A practicing surgeon and a researcher in the area of regenerative medicine, fifteen applications of technologies developed Dr. Atala’s laboratory have been used clinically. He is Editor of 25 books and 3 journals, has published over 800 journal articles, and has received over 250 national and international patents. Dr. Atala was elected to the Institute of Medicine of the National Academies of Sciences, to the National Academy of Inventors as a Charter Fellow, and to the American Institute for Medical and Biological Engineering.
Dr. Atala is a recipient of the US Congress funded Christopher Columbus Foundation Award, bestowed on a living American who is currently working on a discovery that will significantly affect society; the World Technology Award in Health and Medicine, for achieving significant and lasting progress; the Edison Science/Medical Award for innovation, the R&D Innovator of the Year Award, and the Smithsonian Ingenuity Award for Bioprinting Tissue and Organs. Dr. Atala’s work was listed twice as Time Magazine’s Top 10 medical breakthroughs of the year, and once as one of 5 discoveries that will change the future of organ transplants. He was named by Scientific American as one of the world’s most influential people in biotechnology, by U.S. News & World Report as one of 14 Pioneers of Medical Progress in the 21st Century, by Life Sciences Intellectual Property Review as one of the top key influencers in the life sciences intellectual property arena, and by Nature Biotechnology as one of the top 10 translational researchers in the world.
Dr. Atala has led or served several national professional and government committees, including the National Institutes of Health working group on Cells and Developmental Biology, the National Institutes of Health Bioengineering Consortium, and the National Cancer Institute’s Advisory Board. He is a founding member of the Tissue Engineering Society, Regenerative Medicine Foundation, Regenerative Medicine Manufacturing Innovation Consortium, Regenerative Medicine Development Organization, and Regenerative Medicine Manufacturing Society.
The first humans emerged on Earth about 4 million years ago, but new evidence from the study of human evolution has revealed compelling evidence that a small group of these hominins was genetically modified by ancient alien visitors to create the first Homo sapiens.
Researcher and author Daniella Fenton has thoroughly analyzed humanity’s earliest origins and its sudden acceleration in brain development nearly 800,000 years ago, and this research has led to a major revelation.
“Homo sapiens is the creation of ancient astronauts who came through a wormhole in the Pleiades star cluster more than 780,000 years ago.”
The Australian researcher, an expert in equine lineages and gene expression, discovered numerous genetic changes that mark humans as abnormal when compared to modern primate species, some so extreme that they are best explained by advanced genetic engineering.
“The potential to deliver ‘one shot cures’ is one of the most attractive aspects of gene therapy, genetically-engineered cell therapy and gene editing. However, such treatments offer a very different outlook with regard to recurring revenue versus chronic therapies,” analyst Salveen Richter wrote in the note to clients Tuesday. “While this proposition carries tremendous value for patients and society, it could represent a challenge for genome medicine developers looking for sustained cash flow.”
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Goldman Sachs warns sales from the most successful disease treatments are difficult to maintain.
When we think about gene editing, the first thing we remember is the designer babies, and that it’s usually called unethical. But actually, gene editing (CRISPR) may be one of the most promising upcoming medical technologies. Learn why in this video.
0:00 — Opening scene. 0:20 — Gene editing is promising. Here’s why. 2:35 — Also, it can transform the beauty industry. 3:49 — How does gene editing work? 4:16 — My thoughts on that. 5:16 — End credits.
OUTLINE: 0:00 — Introduction. 1:27 — Lab leak. 1:00:01 — Gain-of-function research. 1:09:32 — Anthony Fauci. 1:19:14 — Francis Collins. 1:23:56 — Joe Rogan, Brett Weinstein, and Sam Harris. 1:53:53 — Xi Jinping. 2:08:24 — Patient Zero. 2:21:38 — WHO 2:45:28 — Government transparency. 3:07:28 — Likelihood of a cover-up. 3:09:16 — Future of reproduction. 3:44:55 — Jon Stewart. 3:50:14 — Joe Rogan and Sanjay Gupta. 4:15:19 — Ultramarathons. 4:25:21 — Chocolate. 4:33:34 — One Shared World. 4:48:37 — Hope for the future.
