A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene therapy. The study, ...
Adeno-associated virus vector containing an adenine base editor provides an effective strategy to treat hereditary hearing loss caused by the R75W mutation. Moreover, this gene editing technology can ...
- Oral presentation at ASGCT showcases breakthrough technology enabling precise in vivo genome editing in the CNS using a single AAV vector - Two poster presentations describe methods that support ...
Recombinant adeno-associated virus (AAV) vectors are predominantly nonintegrating, but rare genomic integration events have been associated with oncogenesis in neonatal murine models. Here we report a ...
The first peer-reviewed journal in the field of human gene therapy, providing all-inclusive coverage of the research, methods, and clinical developments that are driving today's explosion of gene ...
Adeno‑associated virus (AAV) vectors offer safety and durable expression but lack tumor specificity. This hypothesis proposes ...
BOSTON — A team at Children’s Hospital of Philadelphia (CHOP) led by Rebecca Ahrens-Niklas, MD, PhD, and Lindsey George, MD, has described a case of a brain tumor linked to a rare integration of adeno ...
While the overwhelming scope of tragic outcomes from HIV infection at the origin of the AIDS epidemic are in the past, those living with HIV still require daily treatments. One option includes ...
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Uncovering the principle by which DNA replication initiation sites are determined in the human genome
When cells proliferate, genomic DNA is precisely duplicated once per cell cycle. Abnormalities in this DNA replication process can cause alterations in genomic DNA, promoting cellular aging, cancer, ...
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