Lifeboat Foundation

Many genetic diseases are caused by diverse mutations spread across an entire gene, and designing genome editing approaches for each patient’s mutation would be impractical and costly.

Investigators at Massachusetts General Hospital (MGH) have recently developed an optimized method that improves the accuracy of inserting large DNA segments into a genome.

This approach could be used to insert a whole normal or “wild-type” replacement gene, which could act as a blanket therapy for a disease irrespective of a patient’s particular mutation.

Scientists continue to expand the technological frontiers of CRISPR, along with its enormous potential, in areas ranging from human health to global food supplies. Such is the case with CRISPR-based gene drives, a genetic editing tool designed to influence how genetic elements are passed from one generation to the next.

Gene drives designed for mosquitoes have the potential to curb the spread of malarial infections that cause hundreds of thousands of deaths each year, yet safety issues have been raised because such drives can spread quickly and dominate entire populations. Scientists have explored the principles governing the spread of gene-drive elements in targeted populations such as mosquitoes by testing many different combinations of components that constitute the drive apparatus. They have found, however, that there’s still more to explore and that key questions remain.

In the journal Nature Communications, University of California San Diego researchers led by former Postdoctoral Scholar Gerard Terradas, together with Postdoctoral Scholar Zhiqian Li and Professor Ethan Bier, in close collaboration with UC Berkeley graduate student Jared Bennett and Associate Professor John Marshall, describe the development of a new system for testing and developing gene drives in the laboratory and safely converting them into tools for potential real-world applications.

Scientists have developed a wireless, battery-free implant capable of monitoring dopamine signals in the brain in real-time in small animal models, an advance that could aid in understanding the role neurochemicals play in neurological disorders.

The device, detailed in a study published in ACS Nano, activates or inhibits specific neurons in the brain using light, a technique known as optogenetic stimulation. It also records dopamine activity in freely behaving subjects without the need for bulky or prohibitive sensing equipment, said John Rogers, Ph.D., the Louis Simpson and Kimberly Querrey Professor of Materials Science and Engineering, Biomedical Engineering and Neurological Surgery, and a co-author of the study.

“This device allows neuroscientists to monitor and modulate brain activity in real-time and in a programmable fashion, in mice—a very important class of animal model for neuroscience studies,” Rogers said.

In this video I showcase a program that I have been working on for simulating evolution by natural selection. I dive into various mechanisms of the simulation and go over some interesting real-life biology in the process. The key aim of this project is to evolve multicellular organisms, starting from single-celled protozoa-like creatures that must collect mass and energy from their surroundings in order to survive, grow and reproduce.

Chapters:
00:00 — Introduction.
00:56 — Life of a protozoan.
02:46 — The start of the simulation.
05:57 — How the cells work.
06:53 — Introducing multicellular colonies.
08:33 — Understanding evolution.
11:38 — Looking at data from the simulation.
13:27 — Evolving epigenetics introduction.
14:14 — Waddington’s Landscape and cell specialisation.
15:22 — The Central Dogma of Molecular Biology.
16:05 — Gene Regulatory Networks.
16:54 — Outro.
17:30 — Watching the simulation.

Continue reading “Simulating Cellular Evolution: The Path To Multicellularity” »

Since the success of the COVID-19 vaccine, RNA therapies have been the object of increasing interest in the biotech world. These therapies work with your body to target the genetic root of diseases and infections, a promising alternative treatment method to that of traditional pharmaceutical drugs.

Lipid nanoparticles (LNPs) have been successfully used in drug delivery for decades. FDA-approved therapies use them as vehicles for delivering messenger RNA (mRNA), which prompts the cell to make new proteins, and small interfering RNA (siRNA), which instruct the cell to silence or inhibit the expression of certain proteins.

The biggest challenge in developing a successful RNA therapy is its targeted delivery. Research is now confronting the current limitations of LNPs, which have left many diseases without an effective RNA therapy.

Combining a map of gene regulatory sites with disease-associated loci has uncovered a new genetic risk factor of adult-onset macular degeneration (AMD), according to a new study publishing January 17 in the open access journal PLOS Biology by Ran Elkon and Ruth Ashery-Padan of Tel Aviv University, Israel, and colleagues. The finding advances the understanding of the leading cause of visual impairment in adults.

AMD is caused by dysfunction in the retinal pigmented epithelium (RPE), a layer of tissue sandwiched between the photoreceptors that receive light, and the choriocapillaris, which nourishes the retina. Because of the central importance of the RPE in AMD, the authors began by exploring a transcription factor (a protein that regulates specific genes) called LHX2 which, based on the team’s analysis of mouse mutants, is central to RPE development. Knocking down LHX2 activity in RPE derived from human stem cells, they found that most affected genes were down-regulated, indicating that LHX2’s role was likely that of a transcriptional activator, binding to regulatory sites on the genome to increase activity of other genes.

The authors found that one affected gene, called OTX2, collaborated with LHX2 to regulate many genes in the RPE. By mapping the genomic sites that OTX2 and LHX2 could bind to, they showed that 68% of those that bound LHX2 were also bound by OTX2 (864 sites in all), suggesting they likely work together to promote the activity of a large suite of genes involved in RPE development and function.

Summary: Study uncovers new genetic risk factors for age-related macular degeneration, a leading cause of vision loss in adults.

Source: PLOS

Combining a map of gene regulatory sites with disease-associated loci has uncovered a new genetic risk factor of adult-onset macular degeneration (AMD), according to a new study publishing January 17 in the open-access journal PLOS Biology by Ran Elkon and Ruth Ashery-Padan of Tel Aviv University, Israel, and colleagues.

Researchers at Chalmers University of Technology have made a ground-breaking discovery in the field of synthetic DNA, using AI to control the cells’ protein production.

This new technology could revolutionize the way we produce vaccines, drugs for severe diseases, and alternative food proteins by making the process faster and significantly cheaper than current methods.

The process of gene expression is fundamental to the function of cells in all living organisms. In simple terms, the genetic code in DNA is transcribed into the molecule messenger RNA (mRNA), which tells the cell’s factory which protein to produce and in what quantities.

A study by the HSE Centre for Language and Brain has confirmed the role of the corpus callosum in language lateralization, the distribution of language processing functions between the brain’s hemispheres. The authors came up with an innovative language task for their study subjects and applied advanced neuroimaging methods to the data collected. A paper on their findings has been published in PLoS ONE.

Functional asymmetry between the two cerebral hemispheres in performing higher-level cognitive functions is a major characteristic of the human brain. For example, the left hemisphere plays a leading role in language processing in most people. However, between 10% and 15% of the human population also use the right hemisphere to varying degrees for the same task.

Traditionally, language lateralization to the right hemisphere was explained by handedness, as it is mainly found in left-handed and ambidextrous (using both hands equally well) individuals. But recent research has demonstrated a genetic difference in the way language is processed by left-handed and ambidextrous people. In addition to this, some right-handed people also involve their right hemisphere in language functions.