Toggle light / dark theme

Gene therapy uses SIRT6 variant found in centenarians

“So many genes are involved in DNA maintenance, FOXO3 for example, which is very interesting, but it cannot be a therapeutic target because it will trigger a lot of other things,” he explains. “SIRT6 is coding for only one protein and, because it’s a small protein, the cargo size is not too big and it can be easily delivered into cells, so it’s possible to use it as a gene therapy target.”

Some of the other factors that play in Genflow’s favour, says Leire, are that the world has reached a better understanding of the biology of aging, but also that gene therapy has also progressed well over the years.

Scientists Discover That Trees Have A “Heartbeat” Too

It seems everyday more and more information is being uncovered about trees and the many mysteries within them. We know that they are alive, but it seems they are even more alive than we may have thought. Trees are interconnected underground, we also now know that trees can communicate with one another, but recently scientists have discovered that trees actually have a sort of heartbeat, it is just so slow that they’ve never noticed before.

Up until recently scientists had thought that water moved through trees by the process of osmosis, in a sort of continuous matter, but now they’ve discovered that the trunks and branches of the trees are actually contracting and expanding and essentially pumping water up from the roots to the leaves, kind of like how our heart pumps blood throughout our bodies.

— Our Journalism Is Moving — Our investigative journalism and reporting is moving to our new brand called The Pulse. Click here to stay informed.

What AI researchers can learn from the self-assembling brain

But one idea that hasn’t gotten enough attention from the AI community is how the brain creates itself, argues Peter Robin Hiesinger, Professor of Neurobiology at the Free University of Berlin (Freie Universität Berlin).

In his book The Self-Assembling Brain, Hiesinger suggests that instead of looking at the brain from an endpoint perspective, we should study how information encoded in the genome is transformed to become the brain as we grow. This line of study might help discover new ideas and directions of research for the AI community.

The Self-Assembling Brain is organized as a series of seminar presentations interspersed with discussions between a robotics engineer, a neuroscientist, a geneticist, and an AI researcher. The thought-provoking conversations help to understand the views and the holes of each field on topics related to the mind, the brain, intelligence, and AI.

Rocket Relaunches Gene Therapy Program as FDA Lifts Clinical Hold

New gene therapy in trials for Danon disease.

“The only available treatment option for Danon disease is a heart transplant. Currently, there are no specific therapies available for the treatment of Danon disease.”


The U.S. Food and Drug Administration (FDA) lifted the clinical hold it placed on Rocket Pharmaceuticals’ experimental gene therapy for Danon disease. Patient enrollment in the Phase I study will resume, the company announced this morning.

New Jersey-based Rocket Pharmaceuticals said it intends to resume the Phase I program as quickly as possible. Dosing of patients in the pediatric cohort that was receiving the lowest-level of the medication will resume in the third quarter.

The FDA placed the clinical hold on Rocket’s RP-A501 in May of this year. The regulatory agency halted the study to ensure the company modified the study protocol, as well as other supporting documents that included revised guidelines for patient selection and safety management. The trial was not halted due to concerns over safety of the gene therapy. Rocket noted that no drug-related safety events have been observed in cohorts that received low and high doses.

A New Theory of Life’s Multiple Origins

Summary: In order to understand life’s full range of forms, new theoretical frameworks must be developed, researchers say.

Source: Santa Fe Institute.

The history of life on Earth has often been likened to a four-billion-year-old torch relay. One flame, lit at the beginning of the chain, continues to pass on life in the same form all the way down. But what if life is better understood on the analogy of the eye, a convergent organ that evolved from independent origins? What if life evolved not just once, but multiple times independently?

Space Medicine, Health and MedTech Innovations, a lecture

By Susan Ip-Jewell## **Space Medicine, Health and MedTech Innovations, a lecture by Susan Ip-Jewell**

In the frame of the new Space Renaissance Academy Webinar Series programme, chaired by the optimum Sabine Heinz, a quite interesting and rich lecture was given yesterday by Dr. Susan Ip Jewell.

Susan is CEO and founder of Mars Moon Astronautic Academy Research Science (MMAARS), one of the SRI VicePresidents and a pasionate space activist. And she’s Commander of Analog Training missions on Moon and Mars simulated surface.

In her lecture, she gives us a wide overlook on many aspects of human health in space, the edge of the space medicine, the innovative techniques using incremental technologies, developing systems integrating robotic, artificial intelligence, remote telemedicine, avatars and drones.

Btw, Sabine, in addition to being an efficient organizer and coordinator, has revealed unexpected talents as a great media presenter!

Sabine was fantastic, moderating the intense discussion that followed the lecture, about the many challenges humanity is facing, while kicking off the civilian space development.

CRISPR Development Makes Stem Cells “Invisible” to Immune System Without Immunosuppressants

Quick vid and a reminder of the 4th conference of Lifespan.io is this weekend.


Gene editing can make stem cells invisible to the immune system, making it possible to carry out cell therapy transplants without suppressing the patients’ immune response. Scientists in the US and Germany used immune engineering to develop universal cell products that could be used in all transplant patients. The idea is to create stem cells that evade the immune system; these hypoimmune stem cells are then used to generate cells of the desired type that can be transplanted into any patient without the need for immunosuppression, since the cells won’t elicit an immune response. They used CRISPR-Cas9 to knock out two genes involved in the major histocompatibility complex, which is used for self/non-self discrimination. They also increased the expression of a protein that acts as a “don’t eat me” signal to protect cells from macrophages. Together, these changes made the stem cells look less foreign and avoid clearance by macrophages. The team then differentiated endothelial cells and cardiomyocytes from the engineered stem cells, and they used these to treat three different diseases in mice. Cell therapy treatments using the hypoimmune cells were effective in rescuing hindlimbs from vascular blockage, preventing lung damage in an engineered mouse model, and maintaining heart function following a myocardial infarction. Immunosuppression poses obvious risks to a patient, and generating custom cells for transplant therapy is often prohibitively expensive. The development of universal donor cells that can be used as therapeutics could bring the cost down significantly, making cellular therapeutics available to many more patients in a much safer way.

SOURCES AND FURTHER READING
⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺
Engineering Cells to Avoid Immune Detection in Transplants: https://www.lifespan.io/news/engineering-cells-to-avoid-immu…ansplants/

“Hypoimmune induced pluripotent stem cell–derived cell therapeutics treat cardiovascular and pulmonary diseases in immunocompetent allogeneic mice” paper: https://www.pnas.org/content/118/28/e2022091118

LSN episode on Intellia Therapeutics’ clinical trial of NTLA-2001 — https://youtu.be/WKOPTfGqMPA

A New Way to Study Neurodegenerative Diseases

The material properties of these protein droplets are important because they play pivotal roles in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and Alzheimer’s and Parkinson’s diseases. The basic idea is that liquid droplets of certain proteins can change to clogs, or aggregates of molecules, which are hallmarks of these diseases.


Summary: Researchers have developed a new technique to quantify protein droplets associated with a range of neurodegenerative diseases, including Alzheimer’s, Parkinson’s, and ALS.

Source: Rutgers University

Some proteins in cells can separate into small droplets like oil droplets in water, but faults in this process may underlie neurodegenerative diseases in the brains of older people. Now, Rutgers researchers have developed a new method to quantify protein droplets involved in these diseases.

The novel technique, which simultaneously quantifies the surface tension and viscosity, or thickness, of protein droplets, will help scientists to study how they change, opening the way to improved understanding of the mechanisms of these diseases and the development of drug treatments.

CRISPR gene editing: The key benefits (and risks) of modifying our natural world

What’s confusing is that some of the modifications we’re now considering could have been achieved years ago through traditional methods, so our views depend on what we think about the safety of new editing technologies, but also how desperate we are to address environmental degradation.


A process that began centuries ago with selective breeding has developed into genetic modification. We explore the consequences of these controversial tools.

‘A new low’: TRICARE cuts services for children with autism, concerning military families

In March, the Defense Health Agency, which oversees TRICARE, announced that by May, advanced behavioral analysis services outside of clinical settings will no longer be covered by the military insurance.


Registered behavior technicians help implement treatment and behavior plans that teach behaviors and skills universally used.

From April: Autism services for military families could be cut under DoD plan

Prior to the TRICARE changes, technicians could accompany children with autism to school and help facilitate the child’s learning.

/* */