Recombinant Adeno-associated virus (rAAV) is one of the main delivery vectors for gene
therapy. To assess immunogenicity, toxicity, and features of AAV gene therapy in clinical …

Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

Most genetic diseases arise from recessive point mutations that require correction, rather
than disruption, of the pathogenic allele to benefit patients. Base editing has the potential to …

Adeno-associated viruses (AAVs) are small, nonenveloped single-stranded DNA viruses
that require helper viruses to facilitate efficient replication. Despite the presence of humoral …

Gene therapy has a history of controversy. Encouraging results are starting to emerge from
the clinic, but questions are still being asked about the safety of this new molecular …

This study examines transgene expression and biodistribution of adeno-associated virus
(AAV) pseudotyped 1–9 after tail vein (TV) injection in male mice. Using a cytomegalovirus …

Abstract Adeno-associated virus (AAV)-based vectors are promising tools for gene
therapeutic applications, in part because AAVs are nonpathogenic viruses, and vectors …

Recombinant adeno-associated viral (AAV) vectors have rapidly advanced to the forefront of
gene therapy in the past decade. The exponential progress of AAV-based vectors has been …

Duchenne muscular dystrophy (DMD) has been a major target for gene therapy
development for nearly 30 years. DMD is among the most common genetic diseases, and …

The serotypes of adeno-associated virus (AAV) have the potential to become important
resources for clinical gene therapy. In an effort to compare the role of serotype-specific virion …