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Potential new treatment for mast cell cancers reduces tumor size and prevents organ infiltration

A potential new treatment for mast cell cancers reduces the number of mast cells by "mutating" the messenger RNA (mRNA) before it can deliver instructions for manufacturing the gene responsible for cell proliferation.

10 Aug 2021

New study describes SARS-CoV-2 ExoN enzyme that promotes antiviral resistance

A new study describes the structure of a crucial enzyme present in SARS-CoV-2.

8 Aug 2021

Researchers gain new insights into the pathology of Usher syndrome

Human Usher syndrome (USH) is the most common form of hereditary deaf-blindness. Sufferers can be deaf from birth, suffer from balance disorders, and eventually lose their eyesight as the disease progresses.

12 Jul 2021

Study explores the effects of SARS-CoV-2 (and other betacoronaviruses) on cellular mRNA processing

A new study has been released on the bioRxiv* preprint server, which deals with a comprehensive survey on alternative splicing concerning SARS-CoV-2 infection. This study has also included two other betacoronavirurses (SARS-CoV and MERS), a control set of unrelated viruses, and a bacterium, namely, Streptococcus pneumonia. The study findings suggest that several cellular parameters are affected owing to a betacoronavirus infection, which in turn affects some genes and invariably various biological functions.

6 Jul 2021

New components for oligonucleotide drugs show safety, efficacy in treating spinal muscular atrophy

Skoltech researchers and their colleagues from Russia and the UK investigated the safety and efficacy of new chemistry in antisense oligonucleotides used to treat spinal muscular atrophy (SMA), a debilitating genetic disease.

29 Jun 2021

Leducq Foundation provides $7 million to support Cardiac Splicing as a Therapeutic Target project

Diverse messenger RNAs are produced in cells by “alternative splicing.” The Leducq Foundation is now supporting a transatlantic network dedicated to investigating this process in heart muscle cells and how changes in this process contribute to disease. Professor Michael Gotthardt of the MDC and Professor Leslie Leinwand of the University of Colorado Boulder are coordinating the project.

14 Jun 2021

Nanopore sequencing contradicts human genome integration of SARS-CoV-2

Now a new preprint research paper posted to the bioRxiv* server dismisses the possibility, raised by that study, that the virus inserts its genetic material into the host cell’s genome.

2 Jun 2021

Could a mathematical application of information theory identify and predict SARS-CoV-2 mutations?

An interesting new study by researchers at the University of Portsmouth, UK, describes the use of a mathematical method to sequence genomes based on information theory. The method offers an alternative to clinical techniques, allowing mutations to be detected and possibly even predicted. In this way, it opens new research opportunities in bioinformatics and genetics.

31 May 2021

New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy

UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients.

3 May 2021

Research demonstrates multivalent nanobodies overcome SARS-CoV-2 variant mutations

In a recent bioRxiv* preprint paper, researchers isolated anti-RBD nanobodies from llamas and “nanomice” (they engineered to produce VHHs cloned from alpacas, dromedaries and camels).

10 Mar 2021

Researchers identify the trigger of key cellular change in amyotrophic lateral sclerosis

Scientists at the Francis Crick Institute and UCL have identified the trigger of a key cellular change in amyotrophic lateral sclerosis (ALS), a type of motor neurons disease.

4 Mar 2021

Diverse patient population helps identify gene variant associated with childhood asthma

Researchers at Henry Ford Health System, as part of a national asthma collaborative, have identified a gene variant associated with childhood asthma that underscores the importance of including diverse patient populations in research studies.

27 Feb 2021

FDA approval provides targeted treatment option for patients with rare DMD mutation

Today, the U.S. Food and Drug Administration granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping (Exons are pieces of DNA that provide information for making proteins in a person's genome).

25 Feb 2021

Research finds new evidence about the mechanisms controlling skin repair and regeneration

As the air continues to dry and temperatures drop, the yearly battle against dry hands and skin has officially begun. New research from Northwestern University has found new evidence deep within the skin about the mechanisms controlling skin repair and renewal.

19 Jan 2021

Truncating mutation locations tied to diversity, severity of autism symptoms

One of the most recognizable characteristics of autism is an amazing diversity of associated behavioral symptoms. Clinicians view autism as a broad spectrum of related disorders, and the origin of the disease's heterogeneity has puzzled scientists, doctors, and affected families for decades.

22 Dec 2020

SARS-CoV-2's microRNAs may have promoted its jump to humans

A new study uses machine learning to explore the small microRNAs (or miRNAs) of SARS-CoV-2 that may determine the virus's ability to successfully infect humans by altering the course of transcriptional events.

1 Dec 2020

Researchers identify shared molecular roots between lipid dysfunction and autism

Researchers at Harvard Medical School, Massachusetts Institute of Technology and Northwestern University have identified a subtype of autism arising from a cluster of genes that regulate cholesterol metabolism and brain development.

18 Aug 2020

AI-enhanced precision medicine approach lays groundwork for identification of autism subtype

A novel precision medicine approach enhanced by artificial intelligence (AI) has laid the groundwork for what could be the first biomedical screening and intervention tool for a subtype of autism, reports a new study from Northwestern University, Ben Gurion University, Harvard University and the Massachusetts Institute of Technology.

10 Aug 2020

Large-scale analysis of Alport syndrome reveals the effectiveness of existing treatments

A large-scale analysis of the clinical characteristics of Alport syndrome in Japanese patients has revealed that the effectiveness of existing treatment with ACE inhibitors and/or angiotensin receptor blockers (RAS inhibitors) varies depending on the type of mutation in the syndrome's causal gene (COL4A5).

7 Aug 2020

Unique molecular mechanics can pave way for potential treatments for infections

Unique molecular mechanics allowing parasites to thrive in the guts of one billion people open the door to new treatments that are safe for the host.

6 Aug 2020