Adeno Associated Virus (AAV) for Cell and Gene Therapy
Type
Blog Post
...However, until recently safe delivery of nucleic acid cargo (DNA or RNA) to target cells has been challenging...serious adverse effects, and their immunogenic or carcinogenic profiles have limited their usability in clinical...It is an icosahedral, non-enveloped virus that carries a small, approximately 4.7 kb single stranded genome...single dose of subretinal administration of rAAV2 carrying the human gene RPE65, patients with clinical diagnosis...