A Rice University lab is leading the effort to reveal potential threats to the efficacy and safety of therapies based on CRISPR-Cas9, the Nobel Prize-winning gene editing technique, even when it appears to be working as planned.
Bioengineer Gang Bao of Rice’s George R. Brown School of Engineering and his team point out in a paper published in Science Advances that while off-target edits to DNA have long been a cause for concern, unseen changes that accompany on-target edits also need to be recognized — and quantified… Continue reading.
A new technology that relies on a moth-infecting virus and nanomagnets could be used to edit defective genes that give rise to diseases like sickle cell, muscular dystrophy and cystic fibrosis.
Rice University bioengineer Gang Bao has combined magnetic nanoparticles with a viral container drawn from a particular species of moth to deliver CRISPR/Cas9 payloads that modify genes in a specific tissue or organ with spatial control.
Because magnetic fields are simple to manipulate and, unlike light, pass easily through tissue, Bao and his colleagues want to use them to control the expression of viral payloads in target tissues by activating the virus that is otherwise inactivated in blood… Continue reading.
Scientists have successfully used gene editing to repair 20 to 40 percent of stem and progenitor cells taken from the peripheral blood of patients with sickle cell disease, according to Rice University bioengineer Gang Bao.
Bao, in collaboration with Baylor College of Medicine, Texas Children’s Hospital and Stanford University, is working to find a cure for the hereditary disease. A single DNA mutation causes the body to make sticky, crescent-shaped red blood cells that contain abnormal hemoglobin and can block blood flow in limbs and organs.
In his talk at the annual American Association for the Advancement of Science meeting in Austin Feb. 16, Bao revealed results from a series of tests to see whether CRISPR/Cas9-based editing can fix the mutation. His presentation was part of a scientific session titled “Gene Editing and Human Identity: Promising Advances and Ethical Challenges… Continue reading.
HOUSTON – (Dec. 15, 2014) – Gang Bao will bring a host of new expertise to Rice University’s part in the fight against cancer — and many other diseases — when he joins the faculty March 1.
The highly regarded Robert A. Milton Chair in Biomedical Engineering at Georgia Institute of Technology and Emory University is the latest recruit to move to Houston with $6 million in funding from the Cancer Prevention and Research Institute of Texas (CPRIT).
Bao and his colleagues, nine of whom will join him at Rice, cover a wide range of research linked primarily by their interest in the genetic roots of disease and the promise of nanotechnology and biomolecular approaches to treat them.
Among their ongoing projects, lab members are working on targeted genome modification using engineered nucleases, the development of magnetic nanoparticles for use as contrast agents and for ablation of tumors and the application of fluorescent molecular beacons for specific RNA detection in living cells.
“Dr. Bao has an outstanding track record of center leadership in developing and applying nanomedicine for disease diagnosis and treatment, and is a fantastic addition to the Rice effort in translational nanomedicine,” said Michael Deem, chair of the Department of Bioengineering and the John W. Cox Professor of Biochemical and Genetic Engineering.
Pure cardiac muscle cells, ready to transplant into a patient affected by heart disease.
That’s a goal for many cardiology researchers working with stem cells. Having a pure population of cardiac muscle cells is essential for avoiding tumor formation after transplantation, but has been technically challenging.
Researchers at Emory and Georgia Tech have developed a method for purifying cardiac muscle cells from stem cell cultures using molecular beacons.
Magnets could be a tool for directing stem cells’ healing powers to treat conditions such as heart disease or vascular disease.
By feeding stem cells tiny particles made of magnetized iron oxide, scientists at Emory University and the Georgia Institute of Technology can then use magnets to attract the cells to a particular location in the body after intravenous injection.
The results are published online in the journal Small and will appear in an upcoming issue.
The paper was a result of collaboration between the laboratories of W. Robert Taylor of Emory, and Gang Bao of Georgia Tech. Taylor is professor of medicine and biomedical engineering and director of the Division of Cardiology at Emory University School of Medicine. Bao is professor in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University. Co-first authors of the paper are postdoctoral fellows Natalia Landazuri and Sheng Tong. Landazuri is now at the Karolinska Institute in Sweden.
Physicians and engineers within a new center devoted to pediatric nanomedicine will develop targeted, molecular-sized nanoparticles as part of a unique approach to treating pediatric diseases. Specific focus areas will include pediatric heart disease and thrombosis, infectious diseases, cancer, sickle cell disease and cystic fibrosis.
The Center for Pediatric Nanomedicine (CPN) is the first of its kind in the world.
Directed by Gang Bao, the center will involve researchers from Emory University, the Georgia Institute of Technology and Children’s Healthcare of Atlanta.
“Because nano-scale structures are compatible in size to biomolecules, nanomedicine provides unprecedented opportunities for achieving better control of biological processes and drastic improvements in disease detection, therapy and prevention,” says Bao, the Robert A. Milton Professor of Biomedical Engineering in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University.
The Georgia Tech-led Nanomedicine Center for Nucleoprotein Machines has received an award of $16.1 million for five years as part of its renewal by the National Institutes of Health (NIH). The eight-institution research team plans to pursue development of a clinically viable gene correction technology for single-gene disorders and demonstrate the technology’s efficacy with sickle cell disease.
Sickle cell disease is a genetic condition present at birth that affects more than 70,000 Americans. It involves a single altered gene that produces abnormal hemoglobin — the protein that carries oxygen in the blood. In sickle cell disease, red blood cells become hard, sticky and “C” shaped. Sickle cells die early, which causes a constant shortage of red blood cells. The abnormal cells also clog the flow in small blood vessels, causing chronic pain and other serious problems such as infections and acute chest syndrome.
“Even though researchers know sickle cell disease is caused by a single A to T mutation in the beta-globin gene, there is no widely available cure,” said center director Gang Bao, the Robert A. Milton Chair in Biomedical Engineering in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University. “By directly and precisely fixing the single mutation, we hope to reduce or eliminate the sickle cell population in an individual’s blood stream and replace the sickle cells with healthy red blood cells.”
Georgia Tech and Emory University have received a five-year $14.6 million contract from the National Institutes of Health (NIH) to continue the development of nanotechnology and biomolecular engineering tools and methodologies for detecting and treating atherosclerosis.
Atherosclerosis typically occurs in branched or curved regions of arteries where plaques form because of cholesterol build-up. Inflammation can alter the structure of plaques so they become more likely to rupture, potentially causing a blood vessel blockage and leading to heart attack or stroke.
The award will support the interdisciplinary Center for Translational Cardiovascular Nanomedicine as the second phase of the Program of Excellence in Nanotechnology (PEN), originally established in 2005 with funding from the National Heart, Lung, and Blood Institute of the NIH. This Center integrates the biomedical engineering expertise of Georgia Tech and the cardiology strengths of Emory University’s School of Medicine. The broad and long-term goal of the PEN is to improve the diagnosis and treatment of cardiovascular disease, which is the leading cause of death for men and women in the United States.
“In the last five years, we developed a suite of nanotechnology approaches for diagnosing and treating cardiovascular disease and we have demonstrated their efficacy in terms of potential clinical application,” said Gang Bao, the program’s director and the Robert A. Milton Chair in Biomedical Engineering in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University. “For the next five years, we will focus on translating these technologies into clinical utility and we would like to have some of these nanotechnologies ready for human clinical trials by the end of this five-year period.”
To understand the role of inflammation in cardiovascular and other diseases, it is essential to identify and characterize genes that induce an inflammatory response in the body — and the genes that regulate them.
A study published online this week in the journal Proceedings of the National Academy of Sciences suggests that a gene called Hu antigen R (HuR) plays a critical role in inducing and mediating an inflammatory response in cells experiencing mechanical and chemical stresses. The study was supported by the National Institutes of Health.
The findings may open up new possibilities for developing treatments of metabolic diseases associated with inflammation, such as atherosclerosis. Atherosclerosis typically occurs in branched or curved regions of arteries where plaques form because of cholesterol build-up. Inflammation can alter the structure of plaques so that they become more likely to rupture, causing a blood vessel blockage and leading to heart attack or stroke.
“This is the first systematic study showing that HuR not only responds to external stimuli as a stress-sensitive gene, but it also regulates other stress-sensitive genes,” said senior author Gang Bao, the Robert A. Milton Chair in Biomedical Engineering in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University.