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Viral Vector Platforms for Gene Therapy
Viral Vector Platforms for Gene Therapy
In both the laboratory and clinic, scientists harness viral genetic transfer capabilities to develop gene therapies that modulate cellular function.
In both the laboratory and clinic, scientists harness viral genetic transfer capabilities to develop gene therapies that modulate cellular function.
adeno-associated virus
Journey to the Center of the Ear
Niki Spahich, PhD | Aug 28, 2023 | 5 min read
An aqueduct connecting the brain to the ear may make gene therapy for hearing loss less invasive.
From Concept to Cure: Using AAV in Gene Therapy
The Scientist’s Creative Services Team and Bio-Rad Laboratories | Sep 29, 2022 | 1 min read
With the right tools and techniques, researchers develop safe and effective adeno-associated virus (AAV)-based gene therapies.
Preprints Propose Constellation of Causes for Kids’ Liver Disease
Christie Wilcox, PhD | Jul 25, 2022 | 2 min read
Two independent groups suggest the suite of recent unexplained hepatitis cases may stem from coinfection with an adeno-associated virus and a helper adeno- or herpesvirus, a duo which may be especially virulent in children with a particular genetic variant.
A Model for Better Gene Therapy
PhoenixBio | Apr 5, 2022 | 1 min read
Explore how humanized liver chimeric mouse models smooth the transition between the laboratory and clinical trials.
Empty or Full: Separating Therapeutically Viable Viral Vectors
Bio-Techne | Mar 16, 2022 | 1 min read
How to detect impurities during gene therapy vector production
Optimizing Adeno-Associated Virus (AAV) Manufacturing
The Scientist Creative Services Team in collaboration with OXGENE | Feb 16, 2021 | 2 min read
Scientists wield nature’s power to optimize adeno-associated virus (AAV) production and maximize gene therapy safety.
Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy
Emma Yasinski | Sep 25, 2020 | 5 min read
Adeno-associated viral vectors can deliver gene therapies, but AAV-neutralizing antibodies might prevent the medicines from working.
Gene Therapy Finds a Fertile Home in Ohio
Shawna Williams | Jan 13, 2020 | 8 min read
The midwestern state has quietly laid the groundwork for a biotech hub.
Dog Study Revives Concerns About Virus Used for Gene Therapy
Jef Akst | Jan 6, 2020 | 2 min read
Canines treated with an adeno-associated viral (AAV) vector showed evidence that the therapeutic DNA held within the virus can integrate into the host genome, risking the activation of oncogenes.
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Catherine Offord | Nov 12, 2019 | 2 min read
The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.
Positive Trial Results for Experimental DMD Gene Therapy
Catherine Offord | Jun 20, 2018 | 2 min read
Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.
Severe Toxicity Reported in High-Dose AAV Gene Therapy in Animals
Jim Daley | Jan 31, 2018 | 2 min read
Biotech stocks fell in response to the news.
Gene Therapy Temporarily Reverses Type 1 Diabetes in Mice
Shawna Williams | Jan 4, 2018 | 2 min read
Pancreatic cells engineered to produce insulin did not immediately provoke an immune response.
Ancient Viruses as Gene Therapy Vectors
Ashley P. Taylor | Jul 31, 2015 | 3 min read
Researchers deploy ancestors of today’s adeno-associated viruses to deliver gene therapies without immune system interference.
Reducing Gene Therapy-Related Risk
Kerry Grens | Jan 22, 2015 | 1 min read
In a mouse model of a rare disease, scientists have figured out how to reduce the elevated cancer risk tied to a gene therapy treatment.
Gene Therapy Arrives in Europe
Sabrina Richards | Nov 5, 2012 | 4 min read
The European Commission approves the Western hemisphere’s first gene therapy, aimed at correcting a lipid-processing disorder.