“Polygenic Adaptation”
China’s health ministry has issued draft regulations that will restrict the use of gene editing in humans, just three months after Chinese researcher He Jiankui announced that twin girls had been born with edited genomes. The proposal includes severe penalties for those who break the rules. If approved, scientists say the policy could have gains and drawbacks for research.
Lupus can be a stubborn disease to treat. Although many struck by the autoimmune condition live relatively normal lives, some suffer from kidney failure, blood clots, and other complications that can be deadly. Now, scientists have found that a novel treatment that wipes out the immune system’s B cells cures mice of the condition. Though the work is preliminary, it has excited researchers because it uses a therapy already approved for people with blood cancer.
The strategy is known as chimeric antigen receptor (CAR)-T therapy.
As a geneticist, I feel fortunate to live in the post-genomic era. The sequencing of the human genome has made it possible to make advances in understanding human genetics at an unprecedented pace. Genetic research is changing our understanding of early human migration and offering tantalizing insights into human biology. I have high hopes that we will be able to use these insights to better prevent, treat, and potentially cure diseases.
In November last year, the international scientific community was shocked by the announcement by He Jiankui, a CRISPR scientist, that he and his team at the Southern University of Science and Technology in Shenzhen had created the first “gene-edited babies”. The genetic material of these babies had been edited to make them resistant to HIV, smallpox and cholera.
This event has raised a flurry of reactions and controversies, and also demonstrated some deep ambiguities surrounding the risks and implications of research on genome editing, which could fundamentally change how humans are “fabricated”. Beyond the purely scientific aspect, the question of how to balance potential benefits against the potential negative consequences must consider the acceptability of the risks involved.
Chimeric antigen receptor (CAR) T cells are about as cutting-edge as cancer care gets today. Having demonstrated the ability to eradicate tumor cells in up to 90% of patients with certain blood cancers, these engineered immune cells became the first class of gene therapy to win FDA approval in 2017—with Novartis’ Kymriah getting the nod in August, followed by Kite Therapeutics’ Yescarta in October.3 But Alexander Marson, MD, PhD, knows these sophisticated cells are capable of so much more.
Marson, an immunologist at the University of California at San Francisco, is exploring this potential by using the CRISPR-Cas9 system to introduce precisely targeted genome modifications. The idea is that by adding or deleting specific genomic sequences, one can make these cells more lethal for tumors but also safer for the patient. Marson’s team recently developed a platform called SLICE4—single-guide RNA (sgRNA) lentiviral infection with Cas9 protein electroporation—to perform diverse CRISPR modifications in many cells in parallel, in hopes of rapidly identifying changes that measurably improve CAR T-cell performance. “We’re pretty good at manufacturing the ‘hardware’ of gene edited cells, and we’re continuing to improve that,” says Marson. “The really interesting thing will be what genetic ‘software’ we can put into them.”
Positive Screens Warrant Genetic Counseling, Testing
March 06, 2019 03:17 pm Chris Crawford – Mutations in the breast cancer susceptibility genes BRCA1 and BRCA2 are just one of many factors that can greatly increase a woman's risk of developing certain cancers, such as breast and ovarian cancer. One important step in preventing these cancers is to help women understand their risk.
On Jan. 15, the U.S. Preventive Services Task Force (USPSTF) posted a draft recommendation statement(www.uspreventiveservicestaskforce.org) and draft evidence review(www.uspreventiveservicestaskforce.org) on risk assessment, genetic counseling and genetic testing for BRCA-related cancer in women.
Based on its review of the evidence, the USPSTF recommended that physicians screen women who have family members with breast, ovarian, tubal or peritoneal cancer or who have an ethnicity or ancestry associated with BRCA1 or BRCA2 gene mutations with one of several screening tools designed to identify a family history that may be associated with an increased risk for potentially harmful mutations in these breast cancer susceptibility genes, the draft recommendation said.
Arizona could soon be one of the first states to maintain a massive statewide DNA database.
And if the proposed legislation passes, many people — from parent school volunteers and teachers to real estate agents and foster parents — will have no choice but to give up their DNA.
Under Senate Bill 1475, which Sen. David Livingston, R-Peoria, introduced, DNA must be collected from anyone who has to be fingerprinted by the state for a job, to volunteer in certain positions or for a myriad of other reasons.
The bill would even authorize the medical examiner's office in each county to take DNA from any bodies that come into their possession.
The Department of Public Safety would maintain the collected DNA alongside the person's name, Social Security number, date of birth and last known address.
Any DNA in the database could be accessed and used by law enforcement in a criminal investigation. It could also be shared with other government agencies across the country for licensing, death registration, to identify a missing person or to determine someone's real name.
For just the second time, a patient with AIDS appears to have been cured of the disease after receiving a stem cell transplant from a donor with a genetic mutation that provides resistance to HIV, which causes AIDS. But the mutation is exceedingly rare, raising controversial questions of whether it may be replicated using nascent gene-editing tools like CRISPR/Cas9.
The genome editor CRISPR has transformed many areas of biology, but using this tool to enhance certain varieties of crops such as wheat and corn remains difficult because of the plants’ tough cell walls. Now, a major agricultural company has creatively solved that problem by using pollen from one genetically modified plant to carry CRISPR’s components into another plant’s cells. The solution promises to speed the creation of better and more versatile crops, scientists say.
Dante Labs takes the first step of its ambitious project to integrate high coverage genome sequencing in clinical care in clinics and hospitals.
Dante Labs has announced the first of hundreds of European clinics which will integrate whole genome and whole exome sequencing in standard clinical care, further making personalized medicine a standard within the European health care system.
The clinic, "Villa Letizia," is located in L’Aquila, Italy, and is the selected destination for patients across the entire Mediterranean region. Patients will receive 100X whole exome or 30X whole genome sequencing with premium turnaround time and personalized reports as part of their standard clinical care.
Healthy people in the UK are to be controversially offered genetic testing by the National Health Service (NHS) – for a fee. The plan has stoked concerns among geneticists and clinicians, who question its clinical usefulness for healthy people and worry it could waste NHS resources.
UK health minister Matthew Hancock said seriously ill children and adults with genetic conditions will be offered the service for free, but healthy people will pay and have the option of having their DNA analysed by NHS scientists. This will help predict a patient’s risk of developing various conditions, he said.
China has proposed new rules for gene editing and other “high risk” biomedical technologies. The proposals come three months after a Chinese researcher was widely criticized after claiming he had created the world’s first genetically-edited babies.
A draft of the measures was published this week on the website of China’s National Health Commission. The new rules would cover gene editing, stem cell experiments and biological products created for the human body, the Chinese government-supported Global Times newspaper reported.
CRISPR, the gene editing technique that promises to revolutionize healthcare and medicine, is not perfect. Right from the beginning, there have been concerns that this technique, which makes it easy to select specific areas of the genome and quickly and easily snip out and replace pieces of DNA, may also make unintended cuts in other areas of the genome at the same time. And two new studies, one in mice embryos and the other in rice plants, seem to confirm this.
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“This time last year, the first primates cloned through a nucleus transfer technique made headlines around the world. Now, Chinese researchers have pushed the envelope even further – by breaking a regulatory gene in macaques before cloning them. According to the researchers, cloning genetically altered primates has clear benefits for medical testing. But in the wake of controversy over gene editing on humans, progress in this contentious area could be outpacing ethics.
After the new experiment, five infant macaques born at the Chinese Academy of Sciences' Institute of Neuroscience in Shanghai all share the exact same genes, derived from a fibroblast taken from the skin of a donor monkey. More importantly, they all carry a copy of one specific gene – a version of BMAL1 that had been altered in the donor using CRISPR/Cas9 gene-editing technology.”
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