Range of Methods Used in Generating and Purifying AAV Vectors Vectors derived from adeno-associated virus (AAV) provide promising gene delivery vehicles that can be used effectively in large-scale ...

What Are AAV Vector-Based Therapies? Gene therapies are groundbreaking approaches utilizing viral and non-viral vectors. These vectors introduce therapeutic genes into patient cells to treat diseases, ...

The first vector-delivered gene therapies to receive FDA approval for clinical use were Luxturna,1 Spark Therapeutics’ vision loss cure, and Zolgensma,2 Novartis’ spinal muscular atrophy treatment.

Adeno-associated virus (AAV) vectors have become widely recognized as a fundamental tool in gene therapy, primarily due to their potential as a delivery system for the treatment of genetic disorders.

In a recent review published in Signal Transduction and Targeted Therapy, researchers presented recombinant adeno-associated virus (rAAV)-based genetic applications to treat human diseases. Study: ...

In the mid-1990s, Hiroyuki Nakai, now an adeno-associated virus (AAV) researcher at the Oregon Health and Science University, moved to the United States. Nakai quickly became fascinated with the ...

An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent cells and leading to syndromic hearing loss. In a new experimental 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 ...

PITTSBURGH--(BUSINESS WIRE)--Avista Therapeutics, which recently launched as a spinout of leading health system UPMC, aims to develop innovative gene therapies for rare ophthalmic conditions. The new ...