gene therapy

Gain of function to build therapeutically useful viral vectors

Another excellent example of gain of function research is modification of a viral vector to make it more useful for human gene therapy. Adenovirus associated virus (AAV) is the most commonly used vector for a variety of gene therapy applications, including gene replacement and gene editing. These small viruses, which comprise a single-stranded DNA genome …

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An ancestral vector improves on this year€™s model

Adenovirus associated virus (AAV) vectors are being increasingly used for gene therapy because they are not pathogenic in humans and persist for long periods in certain cell types. Currently 120 gene delivery clinical trials with these vectors are in progress, and two have been approved: Luxturna to treat a rare form of blindness, and another …

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CRISPR-ing HIV-1

By Gertrud U. Rey Although antiretroviral therapy (ART) has been highly effective at controlling HIV-1 viral loads in the bloodstream of infected individuals, the virus remains latent in infected cells and starts replicating within a couple of weeks upon termination of therapy.

TWiV 459: Polio turns over a new leaf

The TWiV team reviews the first FDA approved gene therapy, accidental exposure to poliovirus type 2 in a manufacturing plant, and production of a candidate poliovirus vaccine in plants. Click arrow to play Download TWiV 459 (63 MB .mp3, 105 min) Subscribe (free): iTunes, RSS, email Become a patron of TWiV! Show notes at microbe.tv/twiv

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