Genetic Engineering
Episodes
In this clip, Dr. George Church describes the pros and cons of animal research and how we could move beyond its use with advanced technologies.
In this clip, Dr. George Church discusses how gene editing could uniquely improve human life in the future.
In this clip, Dr. George Church discusses these findings and what they could mean for the future of public health.
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In this clip, Dr. George Church describes the pros and cons of animal research and how we could move beyond its use with advanced technologies.
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In this clip, Dr. George Church discusses how gene editing could uniquely improve human life in the future.
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In this clip, Dr. George Church discusses these findings and what they could mean for the future of public health.
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In this clip, Dr. George Church explains how sleeping and dreaming can inspire scientific ideas and artistic vision.
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In this clip, Dr. George Church discusses the opportunities and possible dangers of tools for genetic engineering.
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In this clip, Dr. George Church discusses how aging might be reversed and what animal research can tell us about human aging.
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In this clip, Dr. George Church discusses how engineering animal organs may make them suitable for human transplant.
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In this clip, Dr. George Church shares insights on the future of gene therapies in healthcare.
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In this clip, Dr. George Church describes how gene writing projects could deliver health benefits for future generations.
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In this clip, Dr. George Church describes Genome Project-Write, its goals, and the technology fueling it.
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In this clip, Dr. George Church discusses how revolutionary technologies such as multiplex genome editing and genome writing could make this and other advances in human health possible.
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In this clip, Dr. George Church discusses the Human Genome Project and how it has revolutionized many areas of science.
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Dr. George Church discusses revolutionary technologies in the field of genetic engineering.
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Methylation and demethylation are critical processes in development and interfering with the enzymes that carry out these two opposing processes can play critical roles in epigenetic age.
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Dr. Steve Horvath found a way to measure biological aging – a type of "clock" – based on the methylation pattern of an organism's genome. This video primer explains the basics of epigenetic clocks, the topic of our interview with Dr. Steve Horvath.
Topic Pages
News & Publications
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Vitamin B12 supports metabolic processes involved in stem cell reprogramming and tissue repair. www.irbbarcelona.org
Proteins called Yamanaka factors can reprogram differentiated (mature) cells into pluripotent stem cells. However, scientists don’t fully understand the metabolic requirements underlying this process. A new study shows that vitamin B12 supports the metabolic processes involved in cellular reprogramming.
First, researchers investigated how gut bacteria influence cellular reprogramming in mice. They induced gene expression to initiate reprogramming, and then they treated the mice with antibiotics to disrupt their gut microbiota. They found that reprogramming efficiency in the colon and stomach decreased markedly, and the gut microbial composition changed, altering vitamin B12 metabolism.
Next, they provided the mice with supplemental vitamin B12. They found that B12 promoted the methylation of histone H3 at a specific site known as H3K36me3, an epigenetic marker that is crucial in preventing the start of improper transcription. Then, they studied the effects of vitamin B12 deficiency in an animal model of ulcerative colitis and found that supplementing with vitamin B12 accelerated tissue repair in the colon.
These findings suggest that vitamin B12 is pivotal in enhancing cellular reprogramming efficiency and promoting tissue repair. They also underscore B12’s importance in fundamental biological processes and point toward potential therapeutic strategies for tissue regeneration and rejuvenation. Learn more about Yamanaka factors and cellular reprogramming in this clip featuring Dr. Steve Horvath.
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Ovarian cancer tumors, even at advanced stages, may be effectively targeted and eliminated by CAR-T immune therapies, a new study in mice suggests. www.sciencedaily.com
CAR-T, or chimeric antigen receptor T-cell therapy, is an immunotherapy approach that involves genetically modifying a person’s own T cells so they can recognize and target specific proteins on cancer cell surfaces, enhancing the immune system’s capacity to seek out and destroy malignant cells. CAR-T therapies have been successful against blood cancers, such as leukemia, lymphoma, or myeloma, but have largely failed with solid tumors. Now, a new study in mice demonstrates that CAR-T is effective against ovarian cancer, nearly doubling survival time.
Researchers identified a unique carbohydrate present only on the surface of solid tumor cells, not healthy ones, and engineered CARs with a strong affinity for the carbohydrate. Then, they delivered the CAR-T therapy via intravenous injection to mice with ovarian cancer. Because ovarian cancer treatments delivered directly into the abdominal area are particularly effective, they also administered the CAR-T therapy into the animals' abdomens.
They found that the CAR-equipped T cells effectively located and eliminated the cancer cells, promoting tumor shrinkage or elimination with just one dose. The genetically engineered cells maintained their effectiveness for several months, with no evidence of toxicity or adverse effects. Intravenous injection of CAR-T cells increased survival to 145 days, but direct delivery into the animals' abdomens extended survival to 270 days.
These findings demonstrate that modified CAR-T cells show promise as a potential treatment for ovarian cancer and other solid tumors. Future studies are needed to assess the treatment’s effectiveness in humans. Learn more about genetic engineering in this episode featuring Dr. George Church.
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‘Gene silencing’ therapy reduces lipoprotein(a), an important risk factor of heart disease, by up to 98% www.sciencedaily.com
From the article:
In the trial, participants who received higher doses of SLN360 – a small interfering RNA (siRNA) therapeutic that “silences” the gene responsible for lipoprotein(a) production – saw their lipoprotein(a) levels drop by as much as 96%-98%. Five months later, these participants' lipoprotein(a) – also known as Lp(a) – levels remained 71%-81% lower than baseline.
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Participants receiving 300 mg and 600 mg of SLN360 had a maximum of 96% and 98% reduction in Lp(a) levels, and a reduction of 71% and 81% at five months compared to baseline. Those receiving a placebo saw no change in Lp(a) levels. The highest doses also reduced LDL cholesterol by about 20%-25%.
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Gene therapy for thalassemia ends need for transfusions in young children www.sciencedaily.com
Gene therapy treats transfusion-dependent beta-thalassemia.
Gene therapy is a technique that modifies a person’s genes to prevent, treat, or cure a disease. Gene therapies work by a variety of mechanisms, including replacing a disease-causing gene with a healthy version of the gene, inactivating a disease-causing gene, or introducing a new or modified gene or genes into the body. Findings from a phase 3 clinical trial suggest that beti-cel gene therapy successfully treats transfusion-dependent beta-thalassemia.
Beti-cel is a gene therapy that works by adding a modified form of the beta-globin gene into a recipient’s own blood cell-producing stem cells. This process relies on transduction, the process by which foreign DNA is introduced into a cell by a virus or viral vector. Beti-cel therapy is designed to facilitate the production of normal, healthy hemoglobin and obviate the need for transfusions.
Thalassemia is a class of inherited blood disorders that affect the genes for hemoglobin, the oxygen-carrying component of the red blood cells. Hemoglobin, which consists of two proteins (alpha and beta) becomes incorporated into red blood cells during their maturation. Insufficient production of either the alpha or beta proteins due to genetic defect impairs oxygen transport via the red blood cells, inducing anemia and often necessitating blood transfusions.
The study involved 22 people (ages 4 to 34 years) with transfusion-dependent beta-thalassemia. Prior to receiving the gene therapy, each of the participants underwent chemotherapy to temporarily halt their red blood cell production. The investigators administered beti-cel intravenously and monitored the recipients for an average of 30 months.
They found that 20 of the 22 participants, including six who were under the age of 12 years, no longer required blood transfusions after receiving the gene therapy, typically within one month of administration. Four of the participants experienced adverse events related to the gene therapy, but most events were mild except for one event in which the participant developed thrombocytopenia, a condition in which blood platelet levels are too low.
These findings suggest that beti-cel gene therapy successfully treats transfusion-dependent beta-thalassemia and opens the door for more gene therapy strategies and applications in the future. Learn more about gene therapy in this episode featuring world-renowned geneticist Dr. George Church.
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Spinal cord injuries typically cause irreversible loss of sensation and function below the site of injury. Approximately 17,000 people living in the United States will experience traumatic spinal cord injury in any given year. Findings from a new study in mice suggest that a designer cytokine can restore spinal cord function.
Cytokines are a broad category of naturally occurring small proteins that are important in cell signaling. They are released by cells and influence the behavior of other cells. Designer cytokines are genetically engineered proteins that perform specific functions. The authors of the study engineered hyper-interleukin-6, a cytokine that has been shown to regrow neurons of the visual system.
The study involved mice that had sustained a spinal cord injury and were paralyzed. The authors of the study used viral gene therapy to induce hyper-interleukin-6 production in the animals' damaged neurons.
They found that the hyper-interleukin-6 production caused the axons of various nerve cells in the brain and spinal cord to regenerate within one week of gene therapy. Within two to three weeks post-procedure, the mice began to walk again.
These findings suggest that delivery of a designer cytokine via gene therapy shows promise as a strategy to restore sensory and functional losses after spinal cord injury in mice.