Adenine

First Mice Engineered to Survive COVID-19 Like Young, Healthy Humans

Retrieved on: 
Wednesday, November 1, 2023

NEW YORK, Nov. 1, 2023 /PRNewswire/ -- Researchers have genetically engineered the first mice that get a human-like form of COVID-19, according to a study published online November 1 in Nature.

Key Points: 
  • NEW YORK, Nov. 1, 2023 /PRNewswire/ -- Researchers have genetically engineered the first mice that get a human-like form of COVID-19, according to a study published online November 1 in Nature.
  • The mice with this genetic change developed symptoms similar to young humans infected with the virus causing COVID-19, instead of dying upon infection as had occurred with prior mouse models.
  • "This has been a major missing piece in efforts to develop new drugs against this virus."
  • Boeke also receives consulting fees and royalties from OpenTrons, and holds equity in the company.

Quantum dots are part of a revolution in engineering atoms in useful ways – Nobel Prize for chemistry recognizes the power of nanotechnology

Retrieved on: 
Wednesday, October 4, 2023

The 2023 Nobel Prize for chemistry isn’t the first Nobel awarded for research in nanotechnology.

Key Points: 
  • The 2023 Nobel Prize for chemistry isn’t the first Nobel awarded for research in nanotechnology.
  • But it is perhaps the most colorful application of the technology to be associated with the accolade.
  • This year’s prize recognizes Moungi Bawendi, Louis Brus and Alexei Ekimov for the discovery and development of quantum dots.
  • Quantum dots brilliantly fluoresce: They absorb one color of light and reemit it nearly instantaneously as another color.

Skip chemical bonds, rely on quantum physics

    • For instance, some of the earliest dyes started with a clear substance such as analine, transformed through chemical reactions to the desired hue.
    • Rather than depending on chemical bonds to determine the wavelengths of light they absorb and emit, they rely on very small clusters of semiconducting materials.
    • Early quantum dots were often based on cadmium selenide for instance – the component materials of which are toxic.
    • And yet, quantum dots are a pivotal part of a technology transition that’s revolutionizing how people work with atoms and molecules.

‘Base coding’ on an atomic level

    • This concept is intuitive when it comes to computing, where programmers use the “base code” of 1,s and 0’s, albeit through higher level languages.
    • This ability to work with base codes also extends to the material world.
    • Here, the code is made up of atoms and molecules and how they are arranged in ways that lead to novel properties.
    • Bawendi, Brus and Ekimov’s work on quantum dots is a perfect example of this form of material-world base coding.

Nobel prize in medicine awarded to mRNA pioneers – here's how their discovery was integral to COVID vaccine development

Retrieved on: 
Monday, October 2, 2023

The rapid development of these vaccines changed the course of the pandemic, providing protection against the SARS-CoV-2 virus.

Key Points: 
  • The rapid development of these vaccines changed the course of the pandemic, providing protection against the SARS-CoV-2 virus.
  • Their discovery was not only integral to COVID-19 vaccine development, but may also lead to the development of many other therapies – such as vaccines for cancer.

Life’s work

    • It’s made in the body from our very own DNA in a process called translation.
    • DNA is our special encoded handbook of instructions for manufacturing proteins, which are the building blocks for material in the body.
    • The cells then make whatever protein they’ve been instructed to, such as haemoglobin for helping red blood cells carry oxygen around the body.
    • The researchers faced two major challenges as they began their work.
    • Karikó and Weissman’s research had successfully eliminated the obstacles that had previously stood in the way of using mRNA clinically.

COVID vaccines

    • Researchers had already been working on developing mRNA vaccines before the pandemic, such as a vaccine for Ebola that didn’t receive much commercial interest.
    • This produced a harmless COVID particle which our cells then replicated, allowing our bodies to protect us from severe COVID infections when it encountered the real virus.
    • Studies have also shown mRNA vaccines might be useful in treating certain types of cancer.

New research reveals that Ötzi the iceman was bald and probably from a farming family – what else can DNA uncover?

Retrieved on: 
Thursday, August 24, 2023

This amazing find would subsequently become known as Ötzi the Iceman.

Key Points: 
  • This amazing find would subsequently become known as Ötzi the Iceman.
  • His body and belongings were extensively studied, prompting numerous questions: what was he doing here?
  • His unique preservation enabled the sequencing of Ötzi’s whole genome – the complete “instruction booklet” for building a human.
  • But it was enough for a team led by Turi King at the University of Leicester to extract fragments of DNA from them.

Crime scene samples

    • Sequencing a genome, which comprises billions of DNA bases, enables scientists to evaluate regions of the human genome that contribute to appearance.
    • For more than 30 years, forensic scientists have looked at specific highly variable regions in DNA to match these to crime scene samples, or to relatives of a suspect or victim.
    • So how likely is it that DNA from such a sample could accurately paint a picture of me?
    • Can forensic scientists build a kind of identikit photo from a crime scene DNA sample?
    • Hair colour can be predicted from DNA, but darker shades of hair are more accurately predicted than blonde hair.

Environmental factors

    • Commercially sold laboratory kits such as Hirisplex can simultaneously evaluate several DNA regions to predict the hair and eye colour from a biological sample.
    • However, unlike eye colour, hair colour prediction from DNA is only of value until midlife, when the natural processes of ageing lead to greying or white hair.
    • These processes also lead to hair loss in some people and more than 300 gene variants have been linked to baldness.
    • More representative data from the rest of the world will therefore enhance studies in forensic archaeology, such as the Ötzi research.

Your genetic code has lots of 'words' for the same thing – information theory may help explain the redundancies

Retrieved on: 
Thursday, July 27, 2023

The universality of the genetic code indicates a common ancestry among all living organisms and the essential role this code plays in the structure, function and regulation of biological cells.

Key Points: 
  • The universality of the genetic code indicates a common ancestry among all living organisms and the essential role this code plays in the structure, function and regulation of biological cells.
  • Understanding how the genetic code works is the foundation of genetic engineering and synthetic biology.
  • Just as computers need strings of binary code to function, biological processes also rely on bits of information.

Different words for the same thing

    • Ribosomes read three-letter words called codons, and there are 64 different possible combinations of the four letters that make different codons.
    • In this list of 64 words, 61 encode amino acids, and three signal the ribosome to stop protein synthesis in the cell.
    • In fact, since there are only 20 amino acids but 61 different words to encode them, there is quite a lot of overlap.

Engineering nature’s guidelines

    • The mapping of the 61 codes onto the the 20 amino acids would be roughly equal, with each amino acid assigned three codons.
    • Not only does the final form of a protein need to be optimal, but so do its intermediate forms.
    • Scientists understand some of the guidelines that nature follows when engineering the genetic code.

Information theory and genetics

    • Nature’s affinity for optimization using this irrational number is responsible for the infinitely repeating fractals seen in jagged shorelines, fern leaves, snowflakes and trees.
    • Beyond biology, information optimization using e also has applications in mathematics and cosmology.
    • Entropy is a measure of disorder in a system, and the maximum entropy principle states that systems evolve to states of greater disorder.
    • Although there are many biological mysteries that scientists have yet to solve, information theory can be a powerful tool to help crack the genetic code.

BioVie Issues Letter to Shareholders

Retrieved on: 
Tuesday, July 18, 2023

Over 3,000 significant correlations were found linking reductions in DNA methylation of various CpGs and cognitive, biomarker and neuroimaging endpoints.

Key Points: 
  • Over 3,000 significant correlations were found linking reductions in DNA methylation of various CpGs and cognitive, biomarker and neuroimaging endpoints.
  • I have prepared this letter to shareholders to provide an update that synthesizes all the information that we have released and presented at recent medical conferences.
  • NE3107 reduced CSF phospho-tau levels by -1.66 pg/mL (p=0.0343) and the ratio of p-tau to amyloid beta 42 (Aβ42) by -0.0024 (p=0.0401).
  • P-tau and Aβ are traditional biomarkers of AD progression and have been the focus for AD researchers for decades.

Keros Therapeutics Presents Clinical Trial and Preclinical Study Results from its KER-050 Program and Preclinical Data from its ALK2 Inhibitor Program at the 28th Annual Congress of the European Hematology Association

Retrieved on: 
Friday, June 9, 2023

In addition, Keros announced preclinical data evaluating activin receptor-like kinase-2 (“ALK2”) inhibition, as well as its combination with RKER-050, as potential treatment options for anemia of inflammation.

Key Points: 
  • In addition, Keros announced preclinical data evaluating activin receptor-like kinase-2 (“ALK2”) inhibition, as well as its combination with RKER-050, as potential treatment options for anemia of inflammation.
  • Of these patients, 37 had completed at least 24 weeks of treatment or discontinued as of the data cut-off date (the “evaluated RP2D patients”).
  • Data for hematological response and markers of hematopoiesis were presented from exploratory analyses of these evaluated RP2D patients.
  • By targeting ALK2 inhibition to suppress hepcidin, RKER-216 increased iron availability for erythropoiesis and partially rescued anemia in CKD mice.

Vesigen Highlights Therapeutic Platform for Non-Viral Delivery of Gene Editors and RNA at ASGCT 2023

Retrieved on: 
Monday, May 22, 2023

Vesigen Therapeutics, Inc., a biotechnology company developing a novel non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, showcased eight data presentations at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting held May 16-20, 2023 in Los Angeles.

Key Points: 
  • Vesigen Therapeutics, Inc., a biotechnology company developing a novel non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, showcased eight data presentations at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting held May 16-20, 2023 in Los Angeles.
  • “Based on the substantial evidence generated to date, we believe our approach enables highly efficient, tunable, and re-dosable delivery of multiple emerging therapeutic modalities.
  • In mouse models, functional delivery of engineered ARMMs was observed in the retina, lung, spleen, liver, and other tissues.
  • Three data presentations, including collaborations with Lonza Cell and Gene Technologies and NanoFCM, highlighted development of scalable approaches to produce and characterize ARMMs.

Wave Life Sciences and GSK Announce Collaboration to Drive Discovery and Development of Oligonucleotide Therapeutics Focusing on Novel Genetic Targets

Retrieved on: 
Tuesday, December 13, 2022

Wave’s PRISM platform is the only oligonucleotide platform offering three RNA-targeting modalities (editing, splicing, and silencing, including siRNA and antisense).

Key Points: 
  • Wave’s PRISM platform is the only oligonucleotide platform offering three RNA-targeting modalities (editing, splicing, and silencing, including siRNA and antisense).
  • Importantly, these modalities incorporate novel chemistry, including PN backbone chemistry and control of stereochemistry, to optimize the pharmacological properties of therapeutic oligonucleotides.
  • Pairing GSK's genetic expertise with the best-in-class PRISM™ platform enables us to accelerate drug discovery for newly-identified targets, by matching target to modality.
  • Wave Life Sciences (Nasdaq: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases.

Wave Life Sciences to Highlight Advancements from PRISM Platform at Upcoming Scientific Congresses

Retrieved on: 
Monday, October 3, 2022

CAMBRIDGE, Mass., Oct. 03, 2022 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced it will highlight oligonucleotide chemistry advancements from the company’s PRISMTM discovery and drug development platform at upcoming scientific conferences. The company will also highlight its A-to-I RNA base editing oligonucleotides (AIMers), including WVE-006 for alpha-1 antitrypsin deficiency (AATD), as well as its RNA interference (RNAi) capabilities. The scientific congresses include the 18th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), taking place October 2-5, 2022, and the European Society of Gene & Cell Therapy 29th Congress (ESGCT), taking place October 11-14, 2022.

Key Points: 
  • Our data at OTS and ESGCT are illustrative of both the evolution and the potential of Waves science, said Chandra Vargeese, PhD, Chief Technology Officer and Head of Platform Discovery Sciences at Wave Life Sciences.
  • PRISM is Wave Life Sciences proprietary discovery and drug development platform that enables genetically defined diseases to be targeted with stereopure oligonucleotides across multiple therapeutic modalities, including silencing, splicing, and editing.
  • Wave Life Sciences (Nasdaq: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases.
  • Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances.