The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

Improved AAV tropism is essential for gene therapy to reach its full potential. New capsid engineering techniques are taking on this challenge. Clinical translation of gene therapies has a history ...

In 2014, researchers combined DNA barcoding with next-generation sequencing (NGS) to develop the adeno-associated virus (AAV) barcode-seq technology for streamlining ...

ddPCR technology then amplifies and detects the ligated oligo pairs, while an ITR-2 assay simultaneously detects the AAV genome. Software analyzes the signals from each capsid type to calculate ...

Mariella is an assistant editor at The Scientist. She has a background in neuroscience, and her work has appeared in Drug Discovery News and Massive Science. View Full Profile. Learn about our ...

By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...

Genome editing and chemical genetics in human cells. A, Adeno-associated virus (AAV) vectors are used to deliver ssDNA directly to the nucleus, which can then engage the homologous recombination ...

Our Bureau, Bengaluru Thursday, March 20, 2025, 17:40 Hrs [IST] ...

Single AAV Systems for HDR-Mediated Genome Editing in Vivo Dual AAV Systems for HDR-Mediated Genome Editing in Vivo ...

Bharat Biotech aims to democratize gene therapies, traditionally considered prohibitively expensive and available primarily ...