Gene therapy has traditionally been applied to well-understood diseases where a single genetic mutation was to blame. A new generation of technology is expanding the potential of gene therapy to treat conditions that were previously unreachable. Since the first gene therapy clinical trials in the 1990s, the technology has made its way into the market for conditions ranging from blindness to cancer. Gene therapy has the potential to fix any genetic mutation causing disease by inserting a new copy of the faulty gene. However, its reach has historically been limited. We’ve been constrained with the things we.
David A. Sinclair, PhD, is a professor in the Department of Genetics at Harvard Medical School and co-director of the Paul F. Glenn Center for the Biological Mechanisms of Aging. Dr. Sinclair’s work focuses on understanding the mechanisms that drive human aging and identifying ways to slow or reverse aging’s effects. In particular, he has examined the role of sirtuins in disease and aging, with special emphasis on how sirtuin activity is modulated by compounds produced by the body as well as those consumed in the diet, such as resveratrol. His work has implications for human metabolism, mitochondrial and neurological health, and cancer.
The first attempt in the United States to use a gene editing tool called CRISPR against cancer seems safe in the three patients who have had it so far, but it’s too soon to know if it will improve survival, doctors reported Wednesday.
The doctors were able to take immune system cells from the patients’ blood and alter them genetically to help them recognize and fight cancer, with minimal and manageable side effects. The treatment deletes three genes that might have been hindering these cells’ ability to attack the disease, and adds a new, fourth feature to help them do the job.
“It’s the most complicated genetic, cellular engineering that’s been attempted so far,” said the study leader, Dr. Edward Stadtmauer of the University of Pennsylvania in Philadelphia. “This is proof that we can safely do gene editing of these cells.”
All children will be able to receive whole genome sequencing at birth, under ambitions laid out by the Health Secretary.
Matt Hancock said that in future, the tests would be routinely offered, alongside standard checks on newborns, in order to map out the risk of genetic diseases, and offer “predictive, personalised” care.
Ministers have already promised that such tests will be offered to all children diagnosed with cancer by the end of this year.
Scientists successfully extended the average lifespan of mice by breeding them using embryonic stem cells with extra-long telomeres. The findings are significant because the researchers managed to extend lifespan without genetic modification, and they also shed light on the aging process and techniques that might someday slow it.
The study — published October 17 in Nature Communications — focuses on telomeres, which are stretches of DNA found at the end of chromosomes.
Because telomeres protect the genetic material inside chromosomes, they’ve been likened to the plastic tips on the ends of shoelaces. But telomeres have also been compared to bomb fuses, or “molecular clocks,” because they become shorter each time a cell divides, eventually shrinking so much that the cell dies or stops dividing. This shortening of our telomeres is associated with aging, cancer, and death.
When the first smartphones arrived, few people understood how they would change our reality. Today, our internet-connected mobile device maps our travel, manages our finances, delivers our dinner, and connects us to every corner of human knowledge. In less than a generation, it has become almost an extension of our central nervous system — so indispensable that we can’t imagine leaving home without it to guide us.
We are about to embark on another journey even more important to every individual and to human society. We are entering the age of genomics, an amazing future that will dramatically improve the health outcomes of people across the planet. Soon, we won’t be able to imagine a time when we left home without knowledge of our genome to guide us.
But this future isn’t a generation away. As early as 2020, I believe we will be living in a world where software uses knowledge of our personal genome to guide us, like a health GPS, toward choices that are appropriate for us as individuals. From the foods we choose to eat to the medicines we take to prevent or cure disease, from helping us avoid exposure to environmental risks to eradicating thousands of genetic diseases, genomics will reveal such immense possibilities that it will feel as if we can see and hear for the first time.
Metabesity 2019: Epigenetic resetting of cellular age mediated by nuclear reprogramming – A new paradigm in overcoming aging and aging-associated diseases. Featuring Vittorio Sebastiano, PhD, Assistant Professor of Stanford University; Co-Founder of Turn Biotechnologies, USA
Researchers at the University of Dundee have made a discovery they believe has the potential to put the brakes on the ‘runaway train’ that is Parkinson’s disease.
The team, based at the Medical Research Council Protein Phosphorylation and Ubiquitylation Unit (MRC-PPU) in the School of Life Sciences, have discovered a new enzyme that inhibits the LRRK2 pathway. Mutations of the LRRK2 gene are the most common cause of genetic Parkinson’s.
Enzymes are molecular machines that regulate the biological processes required to maintain healthy functioning life. They can also be targeted by drugs to increase or decrease the level of certain activity –in this instance the LRRK2 pathway.
Unlike chemotherapy or radiation, which attack cancer directly, CAR-T engineers patients’ immune cells so they can do it themselves. T-cells are removed from the blood and given new genes that produce receptors that let the T-cells recognize and bind to leukemia cells with a specific protein, CD19.
The genetically modified T-cells are then multiplied in the lab and infused back into the patient, where they ideally multiply even further and begin to target and kill cancer cells with CD19.
