Lifeboat Foundation

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.

Continue reading “Rocket Relaunches Gene Therapy Program as FDA Lifts Clinical Hold” »

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?

In order to feed a projected 9 billion people by 2,050 farmers need to grow 50% more food on a limited amount of arable land. As a result, plant scientists are in a race against time to engineer crops with higher yields by improving photosynthesis.

Blue-green algae (cyanobacteria) are known to photosynthesize more efficiently than most crops, so researchers are working to put elements from cyanobacteria into crop plants.

A new study describes a significant step towards achieving that goal. “Absence of Carbonic Anhydrase in Chloroplasts Affects C3 Plant Development but Not Photosynthesis,” published on August 11 2021, in the Proceedings of the National Academy of Sciences.

Summary: Study reveals how the brain analyzes different types of speech which may be linked to how we comprehend sentences and calculate mathematical equations.

Source: SfN

Separate math and language networks segregate naturally when listeners pay attention to one type over the other, according to research recently published in Journal of Neuroscience.

Intelligent systems engineer, STEM advocate, hip-hop artist — ashley llorens, VP, distinguished scientist, managing director microsoft research, microsoft.

Ashley Llorens (https://www.microsoft.com/en-us/research/people/allorens/) is Vice President, Distinguished Scientist & Managing Director, at Microsoft Research Outreach, where he leads a global team to amplify the impact of research at Microsoft and to advance the cause of science and technology research around the world. His team is responsible for driving strategy and execution for Microsoft Research engagement with the rest of Microsoft and with the broader science and technology community, and they invest in high-impact collaborative research projects on behalf of the company, create pipelines for diverse, world-class talent, and generate awareness of the current and envisioned future impact of science and technology research.

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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.

Continue reading “Space Medicine, Health and MedTech Innovations, a lecture” »

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.

Continue reading “CRISPR Development Makes Stem Cells ‘Invisible’ to Immune System Without Immunosuppressants” »

There’s a lot that scientists still don’t understand about pterosaurs, CNET reports, especially because so few fossils have been found. But uncovering this new fossil helps explain how the admittedly terrifying creature lived, hunted, and flourished millions of years ago.

The fossil — a massive jawbone — suggests that the Thapunngaka shawi was a ferocious hunter. The 3.2 foot-long jaw likely held as many as 40 huge, sharp teeth. The creature had a wingspan of 22 feet and a neck longer than a giraffe’s that it used to balance its humongous head, according to CNET.

“This thing would have been quite savage,” study coauthor and University of Queensland School of Biological Sciences PhD student Tim Richards told the site. “It would have cast a great shadow over some quivering little dinosaur that wouldn’t have heard it until it was too late.”

Seeing is believing.