Researchers Are Using siRNAs in New Approach to Fight HIV

Emmanuelle Ho, PhD

Society learned about the value of mRNA during the COVID-19 pandemic when we saw scientists and medical professionals harness its power to deliver a vaccine for the virus within a year. Now, University of Waterloo (Canada) pharmacy associate professor Emmanuel Ho, PhD, has developed a novel nanomedicine loaded with genetic material called small interfering RNAs (siRNAs) to fight human immunodeficiency virus (HIV) using gene therapy. These siRNAs regulate which genes or proteins are turned on or off in our cells and showed a 73 per cent reduction in HIV replication. “This opens the door for new therapeutics in the fight against HIV,” said Dr. Ho, who is among Waterloo’s researchers and entrepreneurs leading health innovation in Canada.

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“Junk DNA” in Birds May Hold Key to Safe, Efficient Gene Therapy

Retrotransposons can insert new genes into a “safe harbor” in the genome, complementing CRISPR gene editing.

Retrotransposons found in the genomes of the white-throated sparrow and the zebra finch are shown to safely shepherd transgenes into the human genome, providing a gene therapy approach complementary to CRISPR-Cas9 gene editing. (Credit: Briana Van Treeck, UC Berkeley).

The recent approval of a CRISPR-Cas9 therapy for sickle cell disease demonstrates that gene editing tools can do a superb job knocking out genes to cure hereditary disease. But it’s still not possible to insert whole genes into the human genome to substitute for defective or deleterious genes. A new technique that employs a retrotransposon from birds to insert genes into the genome holds more promise for gene therapy, because it inserts genes into a “safe harbor” in the human genome where the insertion won’t disrupt essential genes or lead to cancer.

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Enhancing Biomedical Research: Creative Biolabs Unveils Advanced mRNA Delivery Vector Development

Creative Biolabs, a prominent global provider of biotechnology services, has unveiled its state-of-the-art mRNA delivery vector service, aimed at bolstering research and innovation in the biomedicine sector. Dedicated to furnishing top-tier customized solutions to its clientele to address their research and developmental requirements, Creative Biolabs’ mRNA delivery service offers a diverse array of options, including lipid-based vectors, polymer-based vectors, and hybrid vectors, tailored to the varied needs of distinct research endeavors.

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Nebraska Researchers Win NIH Funding for Developing Milk Exosome Technology to Target Specific Cells Related to Disease

Jiantao Guo (left), Professor of Chemistry, and Janos Zempleni, Willa Cather Professor of Molecular Nutrition, were selected as Phase 1 winners in the National Institutes of Health’s Targeted Genome Editor Delivery Challenge. (Credit: University Communication & Marketing, University of Nebraska-Lincoln

A University of Nebraska-Lincoln research duo was named a first-round winner in a National Institutes of Health competition aimed at generating solutions for delivering genome-editing technology to the cells of people with rare and common diseases. Janos Zempleni, PhD, Willa Cather Professor of Molecular Nutrition, and Jiantao Guo, PhD, Professor of Chemistry, were selected as Phase 1 winners in the NIH’s Targeted Genome Editor Delivery Challenge. The challenge is a three-phase competition with prizes totaling $6 million; the University of Nebraska–Lincoln team was among 30 initial recipients announced in December 2023. With the $25,000 prize, Drs. Zempleni and Guo will advance development of universal milk exosomes — natural nanoparticles contained in milk — capable of transporting gene editors to any location in the body.

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Biological Dynamics Has Announced Early Access Program to Its ExoVerita™ Pro Platform for Isolating Exosomes/EVs

Easy-to-use, proprietary technology platform transforms exosome research by automating isolation and accelerating exosome-derived insights into human health and disease

On November 8, 2023, Biological Dynamics, Inc., a leader in exosome-isolation technology, announced the first opportunity for external users to access the company’s ExoVerita Pro, a cost-efficient, automated exosomal enrichment platform that delivers samples with best-in-class yields, purity, and reproducibility. “Technology is revolutionizing our understanding and management of some of our most challenging diseases,” said Paul R. Billings, MD, PhD, CEO and Director of Biological Dynamics. “Data suggests that exosomes play an important role in human health and in decoding diseases. Providing researchers with a more reliable, rapid, and easy-to-use option for capturing exosomes brings us closer to our corporate mission: never diagnosing a disease too late.”

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Study Highlights Importance of Genetic Sequencing to Diagnosis of Syndromic Growth Disorders

Researchers at the University of São Paulo in Brazil analyzed data from 115 children who suffered from conditions considered syndromic (with several associated symptoms) and found a high incidence of overlapping genetic alterations.

In an article published November 21, 2023 in the Journal of Pediatrics, researchers based in Brazil describe the case of a nine-year-old boy admitted to hospital with multiple symptoms and overlapping conditions that made diagnosis difficult, such as short stature, thin tooth enamel (dental enamel hypoplasia), moderate mental deficiency, speech delay, asthma, mildly altered blood sugar, and a history of recurring infections in infancy. The article is titled “Exome Sequencing Identifies Multiple Genetic Diagnoses in Children with Syndromic Growth Disorders.” The team used exome sequencing, in which only the protein-coding portion of the genome is analyzed, to look for genetic mutations, and found them in GCK and BCL11B. As a result, the diagnosis was monogenic diabetes and T-cell abnormality syndrome, both of which are rare diseases. Identification of the exact cause of the problem and the discovery of a blood sugar alteration significantly influenced their choice of treatment. 

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New Ribosome-Targeted Synthetic Antibiotic (Cresomycin) Highly Effective Against Drug-Resistant Bacteria

Molecular model of bacterial ribosome
A new synthetic antibiotic created by Harvard researchers, and colleagues from the University of Illinois-Chicago (UIC), overcomes antimicrobial resistance mechanisms that have rendered many modern drugs ineffective and are driving a global public health crisis. A team led by Andrew Myers, PhD, the Amory Houghton Professor of Chemistry and Chemical Biology at Harvard, reported on February 15, 2024 in Science that their synthetic compound, cresomycin, kills many strains of drug-resistant bacteria, including Staphylococcus aureus and Pseudomonas aeruginosa. The Science article is titled “An Antibiotic Preorganized for Ribosomal Binding Overcomes Antimicrobial Resistance.”

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Rare Disease Day at NIH February 29

Rare Disease Day at NIH will be held at the NIH Main Campus (Natcher Conference Center) on Thursday, February 29, 2024, from 9 am to 5 pm EST. There also will be a virtual livestream via NIH VideoCast, with the event archived for replay afterward. The event agenda will feature panel discussions, rare diseases stories, in-person exhibitors and scientific posters, and an art exhibition. The event is free and open to the public. The preliminary agenda for the event can be seen here. You may register for the event here. Rare Disease Day takes place worldwide, typically on or near the last day of February each year, to raise awareness among policymakers and the public about rare diseases and their impact on patients’ lives. Since 2011, the National Center for Advancing Translational Sciences (NCATS) and the NIH Clinical Center have sponsored Rare Disease Day at NIH as part of this global observance. Rare Disease Day at NIH aims to raise awareness about rare diseases, the people they affect, and NIH collaborations that address scientific challenges and advance research for new treatments.

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Columbia Study Finds Exosome-Based Inhalable Therapy Is a Big Step Forward in Lung Cancer Research

Representation of exosome

Lung cancer is one of the most common cancers and has one of the lowest survival rates in the world. Cytokines, which are small signaling proteins, such as interleukin-12 (IL-12), have demonstrated considerable potential as robust tumor suppressors. However, their applications are limited due to a multitude of severe side effects. In a paper published January 11, 2024 by Nature Nanotechnology, Biomedical Engineering Professor Ke Cheng, PhDand his research group at the Columbia University School of Engineering and Applied Science, together with a collaborator from the University of North Carolina, demonstrate that using nanobubbles, called exosomes, through an inhalation treatment method can directly deliver IL-12 messenger RNA (mRNA) to the lungs. mRNAs are the blueprints for producing specific proteins that participate in a variety of cellular functions. While scientists have previously used liposomes (tiny fat-based particles) or lipid nanoparticles (LNPs) to deliver mRNA, this method has several problems, including a lack of tissue homing, where the particles do not go to the target organs, and concerns about the potential toxicity after long-term exposure. Over the past 15 years, Cheng’s group has been developing exosomes for use as superior drug delivery carriers over liposomes and LNPs in specific indications. The new Nature Nanotechnology article is titled “Inhalable Extracellular Vesicle Delivery of IL-12 mRNA to Treat Lung Cancer and Promote Systemic Immunity.”

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“Zombie Cells” and Health in Aging; Focus of Two Mayo Clinic Studies

Jennifer St. Sauver, PhD

With age, cells can experience senescence, a state where they stop growing but continue releasing inflammatory and tissue-degrading molecules. When a person is young, the immune system responds and eliminates senescent cells, often referred to as “zombie cells.” However, zombie cells linger and contribute to various age-related health problems and diseases. Mayo Clinic researchers, in two studies, shed light on the biology of aging cells.

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