“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.”
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.
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?
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.
Several questions and considerations were raised, by the audience, and by the panelists — Bernard Foing, SRI President, A. V. Autino. former SRI President and SR Academy Strategy Director, Sabine Heinz, Chair of the Webinar Series and of the SR Art Chapter, Thomas Matula, SR Academy, Educational Director — on topics like (randomly): * in few months we will have four civilians flying on ISS: though the media only talk about this exciting event in a superficial mode, several challenges stand in the background. * what is the main danger for health in space, low gravity, radiations, somethimg else. * what will be the embryo development in space? * will reproduction be possible in space? * is there a doctor onboard ISS? * will the civilian visitors to ISS have any kind of medical insurance, or warrants? will anybody be responsible for their life and health? * does any idea exist, about what could be surgery in microgravity? * during civilian space development, will the main danger be the human aspects and behaviours? * will social issues raise and play a meaningful role, during the settlement oin the Solar System? (refer: R. Heinlein, James Corey \.
A lecture by Dr. Susan Ip-Jewell, MMAARS CEO and Founder, Space Renaissance International Vice-President.
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-immune-detection-in-transplants/
“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
LSN episode on CRISPR switching genes on and off — https://youtu.be/WPabduXB7eg.
Science to Save the World video on CRISPR Designer Babies — https://youtu.be/Avi66PtJOOA
LIKE WHAT WE DO? ⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺⎺ If you’d like to help us run this show and/or help Lifespan.io end age-related diseases, you can become a Lifespan Hero: https://lifespan.io/hero. Your support means the world to us!
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.
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.
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.
