The adeno-associated virus (AAV) is a small, nonpathogenic parvovirus, which depends on
helper factors to replicate. Those helper factors can be provided by coinfecting helper …

▪ Abstract Broadly defined, the concept of gene therapy involves the transfer of genetic
material into a cell, tissue, or whole organ, with the goal of curing a disease or at least …

Our ability to manipulate the genetic constitution of the nervous system has come of age with
various technologies, including virus vectors that can efficiently deliver genes to neurons …

Adenoviral, retroviral/lentiviral, adeno-associated viral, and herpesviral vectors are the major
viral vectors used in gene therapy. Compared with non-viral methods, viruses are highly …

US7479554B2 - AAV5 nucleic acids - Google Patents US7479554B2 - AAV5 nucleic acids -
Google Patents AAV5 nucleic acids Download PDF Info Publication number US7479554B2 …

Background: Diseases such as hypertrophic cardiomyopathy (HCM) can lead to severe
outcomes including sudden death. The generation of human induced pluripotent stem cell …

Uncontrolled insertion of gene transfer vectors into the human genome is raising significant
safety concerns for their clinical use. The wild-type adeno-associated virus (AAV) can insert …

US8283151B2 - Isolation, cloning and characterization of new adeno-associated virus (AAV)
serotypes - Google Patents US8283151B2 - Isolation, cloning and characterization of new …

US7419817B2 - Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange
chromatography - Google Patents US7419817B2 - Scalable purification of AAV2, AAV4 or AAV5 …

Ex vivo gene therapy offers a potential treatment for Duchenne muscular dystrophy by
transfection of the dystrophin gene into the patient's own myogenic precursor cells, followed …