article thumbnail

Guide to using AAV vectors in gene therapy

Drug Discovery World

In 2023, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. How is the titer determined?

article thumbnail

Guide to using AAV vectors in gene therapy

Drug Discovery World

Earlier this year, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Study: ‘Hidden’ genes could be key in development of new antibiotics

Scienmag

Membrane-localized phage proteins may also help revitalize, enhance existing antibiotics A study from the Center for Phage Technology, part of Texas A&M’s College of Agriculture and Life Sciences and Texas A&M AgriLife Research, shows how the “hidden” genes in bacteriophages — types of viruses that infect and destroy (..)

Gene 74
article thumbnail

Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

XTalks

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

article thumbnail

Europe: Where is the drug discovery innovation?

Drug Discovery World

Thanks to a prestigious academic legacy, Europe has long been a dominant region for life sciences. Europe is traditionally a life sciences powerhouse, with a strong tradition in pharmaceutical discovery. The oldest, still active pharmaceutical company worldwide, Merck, started life in 1668 in Darmstadt, Germany.

Drugs 59
article thumbnail

CRISPR pioneers Doudna and Charpentier claim Nobel chemistry prize

pharmaphorum

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. Charpentier’s early work in this area led to the discovery of tracrRNA, a previously unknown molecule that defends bacteria from viruses by cleaving their DNA.

article thumbnail

Novartis’ Fabhalta Gets FDA Approval for Rare Complement Blood Disorder

XTalks

PNH is caused by a mutation in the CFB gene that codes for complement factor B, a component of the alternative pathway of complement activation. Mutations in the gene result in the production of red blood cells that are prone to premature destruction (hemolysis).