Lifeboat Foundation

Circa 2021

As described above, molecular therapeutics enabling expression of a truncated dystrophin have been far developed. However, an unprecedented opportunity to correct the disease-causing mutation has arisen with the advent of Crispr-Cas9 technology (Fig. 1).

Since the generation of a Cas9-transgenic mouse [28], which allowed for pinpoint gene alterations specifically in organs targeted by AAVs encoding for the corresponding guide RNAs (gRNAs), it became clear that the inevitable course of inherited diseases might be altered by Cas9-mediated correction. Although certain limitations were unmasked early on, such as the preference of non-homologous end-joining (NHEJ) over homology-directed repair (HDR) upon enzymatic cleavage of the double stranded DNA by Cas9, or the packaging capacity of AAVs, muscular dystrophies seemed an ideal target for genome editing. DMD mutations inducing Duchenne muscular dystrophy (DMD) seemed particularly well suited, since internal truncations of the protein may lead to a shortened but stable protein with partial functional restitution and a milder disease progression, as seen in the allelic Becker muscular dystrophy (BMD).

Continue reading “Genome editing for Duchenne muscular dystrophy: a glimpse of the future?” »

Seattle-based longevity biotech YouthBio Therapeutics has emerged from stealth mode, revealing it is working on the development of “gene therapies aimed at epigenetic rejuvenation, particularly with the help of partial reprogramming by Yamanaka factors.” The company boasts some top longevity science talent, with Dr João Pedro de Magalhães serving as its chief scientific officer and Dr Alejandro Ocampo as lead research collaborator.

Longevity. Technology: Cellular reprogramming is hot, hot hot! YouthBio joins a growing list of companies, including Altos Labs, Shift Bioscience and Turn Bio, among others, all aiming to change the course of human health through this exciting, yet early stage, science. Like everyone else, we’ll be watching all the players very closely – where will the first major breakthrough come from?

Cellular reprogramming is the process by which aged cells can be returned to a pluripotent (embryonic-like) state. This process, which can be achieved using Yamanaka factors, also improves the cells’ aging hallmarks. Partial reprogramming means that Yamanaka factors are induced only for short periods, which is not enough to fully change cells beyond a point of no return but is enough to induce rejuvenation.

By isolating and reprogramming brain cells with dementia-causing genetic mutations, a team at JAX offers a powerful new research tool.

Neuroscientists from St. Petersburg University, led by Professor Allan V. Kalueff, in collaboration with an international team of IT specialists, have become the first in the world to apply the artificial intelligence (AI) algorithms to phenotype zebrafish psychoactive drug responses. They managed to train AI to determine—by fish response—which psychotropic agents were used in the experiment.

The research findings are published in the journal Progress in Neuro-Psychopharmacology and Biological Psychiatry.

The zebrafish (Danio rerio) is a freshwater bony fish that is presently the second-most (after mice) used model organism in biomedical research. The advantages for utilizing zebrafish as a model biological system are numerous, including low maintenance costs and high genetic and physiological similarity to humans. Zebrafish share 70% of genes with us. Furthermore, the simplicity of the zebrafish nervous system enables researchers to achieve more explicit and accurate results, as compared to studies with more complex organisms.

Very good news, if unsurprising.

We already have multiple viable avenues of reducing — or eliminating altogether — this particularly pernicious form of remorseless biological entropy.

So good news… UNLESS you’re one of those people who think death is what MAKES life somehow MEANINGFUL, or that living for thousands of years or more would be BORING.

Continue reading “Mystery of why humans die around 80 may finally be solved” »

Summary: Researchers identified a genetic correlation between blood biomarkers and a range of mental health disorders. The study provides evidence some substance measures within the blood may be involved in the cause of mental illnesses. For example, immune system proteins may be involved in the development of depression, schizophrenia, and anorexia.

Source: The Conversation.

Mental health disorders including depression, schizophrenia, and anorexia show links to biological markers detected in routine blood tests, according to our new study of genetic, biochemical and psychiatric data from almost a million people.

For the first time ever, doctors have successfully transplanted a kidney from a pig to a human — and, they say, the organ functioned normally.

The procedure occurred between a genetically-altered pig and a brain dead human patient at NYU Langone Health, according to The New York Times. The pig was genetically engineered to grow a kidney that would be accepted by a human body. The organ was then attached to the patient’s blood vessels in the upper leg, outside of the abdomen, where the researchers observed it functioning normally.

“It was better than I think we even expected,” Dr. Robert Montgomery, director of the NYU Langone Transplant Institute, told the NYT. Montgomery helped perform the procedure in September and told the paper that it “looked like any transplant I’ve ever done from a living donor. A lot of kidneys from deceased people don’t work right away, and take days or weeks to start. This worked immediately.”

More than 5,000 new virus species have been identified in the world’s oceans, according to a new study.

The study researchers analyzed tens of thousands of water samples from around the globe, hunting for RNA viruses, or viruses that use RNA as their genetic material. The novel coronavirus, for instance, is a type of RNA virus. These viruses are understudied compared with DNA viruses, which use DNA as their genetic material, the authors said.

A variant of the CTLA-4 gene associated with autoimmune disease was found to be more frequent in non-small cell lung cancer (NSCLC) patients who experienced an exceptionally high response to anti-PD-1 immunotherapy and higher immune-related side effects than in a comparable cohort of lung cancer patients and healthy individuals, according to data presented during the AACR Annual Meeting 2022, held April 8–13.

“Inhibitors of the immune checkpoint proteins PD-1/PD-L1 have transformed the cancer treatment landscape. However, there remains large variability in response and unpredictable adverse events, including autoimmune reactions, in NSCLC patients who undergo this treatment,” said presenter India Allen, BSc, from the Garvan Institute of Medical Research, St Vincent’s Medical School, UNSW, Australia. “There are currently limited biomarkers to effectively predict this variability, and the extent to which a patient’s genetic makeup contributes to response is not well understood.”

The occurrence of immune-related adverse events (irAEs)—the side effects that arise in response to activation of the immune system by immunotherapy—is known to correlate with higher response to anti-PD-1 therapy and improved outcomes in NSCLC patients.