This is in stark contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

Amanda Ibe-Enwo is a distinguished researcher at Voyager Therapeutics, a biotechnology company based in Massachusetts, USA.

Stargardt disease, the most common form of juvenile macular degeneration, results from biallelic mutations in the ABCA4 gene. In a recently published study, researchers at the Institute of Molecular ...

You have full access to this article via your institution. This intrinsic property of AAV to undergo intermolecular concatamerization had shown great potential for expanding the current packaging ...

Hansa Biopharma AB, Hansa or the Company today announced its full year and Q4 2024 financial results. Søren Tulstrup, President and CEO, Hansa Biopharma said, "2024 proved to be a transformative year ...

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

OXB (LSE: OXB), a quality and innovation-led cell and gene therapy CDMO, today announces the appointment of Jefferies ...

Correctly measuring AAV vectors, as they are called, is critical to their safety and efficacy. In a recently released study, researchers at the National Institute of Standards and Technology ...

and the US Pharmacopeia—aimed to assess variability across methods of quantifying AAV vectors. Six industry laboratories in the US and Europe took part in the study, which compared values ...

Genethon & Eukarÿs enter strategic partnership to develop a breakthrough technology to reduce the biomanufacturing cost of gene therapies: Evry, France Friday, January 24, 2025, ...