Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy
Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy
Adeno-associated viral vectors can deliver gene therapies, but AAV-neutralizing antibodies might prevent the medicines from working.
Adeno-associated viral vectors can deliver gene therapies, but AAV-neutralizing antibodies might prevent the medicines from working.
hemophilia
The Scientist's LabTalk - Episode 4
The Scientist | Dec 11, 2020 | 1 min read
The Past, Present, and Future of Gene Therapy: How to Scale-up Successfully
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.
Preliminary Results Point to Success of In Vivo Gene Editing
Carolyn Wilke | Feb 12, 2019 | 2 min read
Two studies show signs that the introduced DNA is functioning, but it’s too early to know if patients actually benefit.
Sanofi and Celgene Spend $20 Billion in Major Biotech Acquisitions
Katarina Zimmer | Jan 22, 2018 | 2 min read
Pharmaceutical giant Sanofi to take over hemophilia drugmaker Biovertiv, while Celgene will buy cancer drugmaker Juno Therapeutics.
First In Vivo Human Genome Editing to Be Tested in New Clinical Trial
Abby Olena, PhD | May 18, 2017 | 4 min read
Sangamo Therapeutics will use zinc finger nucleases to introduce the gene for a missing clotting factor into the livers of men with hemophilia B.
Targeting DNA
Jef Akst | Jun 1, 2012 | 10+ min read
After 20 years of high-profile failure, gene therapy is finally well on its way to clinical approval.
Gene Editing Treats Blood Disease
Annie Gottlieb | Jun 27, 2011 | 3 min read
Revising a dysfunctional gene in vivo for the first time, researchers successfully restore blood clotting in hemophiliac mice.