CUTTING ROOM Scientists will soon wield the molecular scissors CRISPR/Cas9 in the human body. Some people with a form of inherited blindness will have the gene editor injected into their eyes, where researchers hope it will snip out a mutation.
Dr. Michael West, CEO of AgeX Therapeutics and Founder of Geron Corporation, discusses breakthroughs in the understanding of biological regeneration and in induced tissue regeneration, through his talk “Hayflick Rewound: Somatic Restriction, Epigenetics, and the Reversibility of Human Aging”. This talk was given at the Ending Age-Related Diseases conference in NYC. Join us at http://lifespan.io/hero
DNA combined with the study of family history has been used to solve high-profile cold cases such as the Golden State Killer. Now, volunteers are using the technique to identify crime victims.
In the future, we may have to deal with biological weapons that target specific groups of people, passing over everyone else.
That’s according to a new report out of Cambridge University’s Centre for the Study of Existential Risk reviewed by The Telegraph. In it, the Cambridge researchers argue that world governments have failed to prepare for futuristic weapons based on advanced technology like artificial intelligence and genetic manipulation — or even a killer pathogen designed to kill only people of a particular race.
Faculty engaged in microbiome research across campus have previously shown that our microbiome plays a key role in defining human health. For example, microbial dysfunction in the infant gut – characterized by the enrichment of particular microbial genes and their products – drive immune dysfunction and can be used to predict the development of allergy and asthma in childhood. Perturbed microbial ecosystems across the human body have been linked to autoimmune disease, metabolic syndromes such as obesity and diabetes, skin diseases, and even multiple sclerosis. Gut microbes can even contribute to metabolizing drugs and influence how much enters the circulation.
Leveraging this expertise and collaborations with UCSF Benioff Children’s Hospitals in Oakland and San Francisco and institutions nationwide, the UCSF Benioff Center for Microbiome Medicine aims to develop a holistic understanding of our earliest interactions with microbes in utero, through birth, and in early life. These efforts aim to find ways of predicting and preventing not only asthma and allergy, but other childhood diseases – including dermatological, gastrointestinal, respiratory and neurological disorders.
“At the same time that we are developing therapeutic strategies to restore microbial ecosystems once they have been damaged,” Lynch said. “We also need to find ways to intervene in at-risk populations in very early life to prevent chronic diseases before they start.”
Three hundred and sixty-six days ago, CeCe Moore woke up to the headline that would change her world: “Suspected Golden State Killer, East Area Rapist Arrested After Eluding Authorities for Decades.” Later that day, those authorities would hold a press conference in front of the Sacramento County District Attorney’s office to explain how, a day earlier, they had finally put handcuffs on the man believed to have committed a series of sadistic rapes and murders that spread terror through California for more than 40 years. But Moore didn’t have to tune in to know how they had done it. “I knew immediately they had cracked it with genetic genealogy and GEDmatch,” she says.
She knew it because at the time, Moore was working as the genetic genealogy researcher on the PBS show Finding Your Roots and had a consulting business helping adoptees find their biological parents. To aid her searches, she regularly logged on to GEDmatch, a public database where hobbyists upload results from consumer genetic testing companies like 23andMe and Ancestry to find relatives with shared DNA and to reverse-engineer their family trees. It had come to her attention that another genealogist on the site, Barbara Rae-Venter, had been uploading files that seemed out of place, and Moore suspected they came not from family members, but from crime scenes. But she had never imagined that one of them belonged to the man believed to be one of the most notorious serial killers in US history. “This was going to be huge,” she remembers telling people that day.
Policing power may be about to get much stronger, thanks to another advance in genetic analysis. A new technique can link the patchy, limited DNA information held in forensic databases to the rich DNA libraries held by family tree-building websites, raising further questions about genetic privacy.
Earlier this year, an ancestry database used by people looking to trace their family history was used to identify the suspected Golden State Killer, a serial killer active in California decades ago. Since his arrest in April, genealogy databases – which allow consumers to upload their DNA sequences – have been used to crack several other cold cases.
The ability to modify multiple genetic elements simultaneously would help to elucidate and control the gene interactions and networks underlying complex cellular functions. However, current genome engineering technologies are limited in both the number and the type of perturbations that can be performed simultaneously. Here, we demonstrate that both Cas12a and a clustered regularly interspaced short palindromic repeat (CRISPR) array can be encoded in a single transcript by adding a stabilizer tertiary RNA structure. By leveraging this system, we illustrate constitutive, conditional, inducible, orthogonal and multiplexed genome engineering of endogenous targets using up to 25 individual CRISPR RNAs delivered on a single plasmid. Our method provides a powerful platform to investigate and orchestrate the sophisticated genetic programs underlying complex cell behaviors.
Today the U.S. Food and Drug Administration announced a comprehensive policy framework for the development and oversight of regenerative medicine products, including novel cellular therapies.
The framework — outlined in a suite of four guidance documents — builds upon the FDA’s existing risk-based regulatory approach to more clearly describe what products are regulated as drugs, devices, and/or biological products. Further, two of the guidance documents propose an efficient, science-based process for helping to ensure the safety and effectiveness of these therapies, while supporting development in this area. The suite of guidance documents also defines a risk-based framework for how the FDA intends to focus its enforcement actions against those products that raise potential significant safety concerns. This modern framework is intended to balance the agency’s commitment to safety with mechanisms to drive further advances in regenerative medicine so innovators can bring new, effective therapies to patients as quickly and safely as possible. The policy also delivers on important provisions of the 21st Century Cures Act.
We’re at the beginning of a paradigm change in medicine with the promise of being able to facilitate regeneration of parts of the human body, where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; new genes can be introduced into the body to combat disease; and adult stem cells can generate replacements for cells that are lost to injury or disease. This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine.